Enroll in 2013 Mentorship Program

Jennifer E. Adair, PhD
Fred Hutchinson Cancer Research Center
*Junior Investigator*

What is your current professional status?

Research Associate

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Nonprofit Research Institute

What is your scientific area of research?

Clinical translation of gene therapy for hematopoietic stem cells.

Why do you want to be a mentor?

Realizing what worked for me may help someone else find direction.

As a mentor, what are you hoping to gain from this experience?

Future research connections, advancing the field by promoting the next generation.             

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 1
            b. Geographic Location - 5
            c. Professional Status / Success - 3 
            d. Compatibility of Career Path - 1
            e. Communication Style - 3

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I am currently a research associate in the laboratory of Hans-Peter Kiem, MD, FACP at the Fred Hutchinson Cancer Research Center planning to transition to an independent research faculty position at an academic or nonprofit research institute or center within the next year. My current work focuses on translating strategies for gene therapy in hematopoietic stem cells into clinical trials including generation of preclinical data, regulatory management and conduct of Phase I and II clinical trials. My research in the lab focuses on development of more efficient and safe strategies for gene therapy with special interest in targeted gene integration and methods to improve engraftment of gene-modified blood cells in a nonmyeloablative transplant setting. I also serve as a member of the New Investigator Committee for the Society.

Click here to view Jennifer Adair's CV.

Mavis Agbandje-McKenna, PhD
University of Florida

What is your current professional status?

Professor of Biochemistry and Molecular Biology

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic Institution: University of Florida, Dept. of Biochemistry - Molecular Biology

What is your scientific area of research?

Structural Virology

Why do you want to be a mentor?

I believe that having a mentor or mentors who can help guide your career development is very important to achieving success in your chosen field of research. I was very fortunate to have been mentored by several senior scientists during my ‘path to professorship.” I had guidance in structure biology, virology, and gene therapy. I do not believe I would be where I am today career wise without this guidance, which is still ongoing from my mentors.

As a mentor, what are you hoping to gain from this experience?

I would like the opportunity to “pay it forward.” My mentors were very selfless in the giving of their time and expertise to teach me about what it means to be a faculty member and to help me successfully compete for grant funding. They also taught me how to maintain a healthy balance between work and family. I think this is very important, especially for women in science. I hope to be able to provide the same type of support/guidance to junior faculty, men and women, alike, especially in these very stressful times of limited funding resources and when everyone seems to be scrambling to keep their job and labs going. I have already started on this path through interactions with several junior faculty members both at my institution and elsewhere across the country.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 1
            b. Geographic Location - 5 (we live in a global world without borders)
            c. Professional Status / Success - 1
            d. Compatibility of Career Path - 5
            e. Communication Style - 3

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

My group uses structure biology approaches (X-ray crystallography and cryo-electron microscopy), combined with biochemistry, biophysics, molecular biology, and in vivo animal studies, to conduct research aimed at improving the efficacy of AAV vectors, a gene delivery system that has shown great promise for clinical therapeutic applications. We aim to elucidate the basic biology of the AAVs, through detailed analysis of the viral capsid protein-protein and protein-DNA interactions as well as viral protein-glycan and host protein interactions which facilitative a successful infection. The lab interacts extensively with the AAV community (national and international) and has become the “go to” lab for AAV capsid structure information for functional annotation. One of our most successful collaborations has been the structure-guided design of the AAV2.5 vector, currently in clinical trials, in collaboration of Professor R. Jude Samulski, UNC Chapel Hill.

Click here to view Mavis Agbandje-McKenna's CV.

Andrew H. Baker, PhD
University of Glasgow

What is your current professional status? 

British Heart Foundation Professor of Translational Cardiovascular Sciences

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic Institution

What is your scientific area of research?

Gene therapy, adenoviruses, cardiovascular disease, miRNA, vascular biology

Why do you want to be a mentor?

I have experience in mentoring. I run the our Institute's mentorship programme and am involved in several schemes. It is an exceptionally important part of training for your scientists.

As a mentor, what are you hoping to gain from this experience?

The reward of advising and helping young scientists make the best decisions for their future.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 2
            b. Geographic Location - 5
            c. Professional Status / Success - 3
            d. Compatibility of Career Path - 1
            e. Communication Style - 4

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

Andrew graduated from the University of London in 1990 with a First Class BSc (Joint Honours) in pharmacology and toxicology and then studied for his PhD with the Leukaemia Research Fund at the University of Wales College Of Medicine, graduating in 1994. He then joined the group led by Professor Andrew Newby for his post-doctoral work in Cardiff and developed adenoviral vectors for gene delivery studies in the cardiovascular system. Dr. Baker then transferred to a lectureship at the University of Bristol (Bristol Heart Institute) to continue studies on adenovirus-mediated gene transfer to assess vascular function in different model systems. In 1999, Dr. Baker joined Professor Anna Dominiczak at the University of Glasgow as a Senior Lecturer in Molecular Medicine, then as Reader and in 2005 as Professor of Molecular Medicine. He is based at the Institute for Cardiovascular and Medical Sciences, which is a translational centre of excellence with a focus on primary and secondary prevention at cardiovascular disease. Andrew was awarded the Blandsford Prize (1990) in pharmacology and the “Update in Thrombolysis Research” (Berlin, 1998) for his publication entitled “Divergent effects of tissue inhibitor of metalloproteinase-1, -2 or -3 overexpression on rat vascular smooth muscle cell invasion, proliferation and death in vitro: TIMP-3 promotes apoptosis” which was published in the Journal of Clinical Investigation. In 1999, he was awarded the British Cardiac Society Young Investigator Research Prize for his work “Gene therapy for vein grafting:  Tissue inhibitor of metalloproteinases-3 (TIMP-3) inhibits neointima formation in vitro and in vivo in part by promoting apoptosis”. He has published over 130 primary research papers.  He was awarded the MakDougall-Brisbane prize from the Royal Society of Edinburgh in 2008 and a fellowship from the Society in 2010.  Also in 2010 he was awarded an Outstanding Achievement Award from the European Society of Cardiology and in 2011 received a Royal Society Wolfson Research Merit Award.  From August 2010 to November 2011 he was Acting Director for the Institute for Cardiovascular and Medical Sciences at the University of Glasgow and in 2011 he was awarded a British Heart Foundation Chair of Translational Cardiovascular Medicine

Click here to view Andrew Baker's' Biosketch.

Renier J. Brentjens, MD, PhD
Memorial Sloan-Kettering Cancer Center
*Junior Investigator*

What is your current professional status? 

I am an  Associate Member & Associate Attending Physician, Leukemia Service, at Memorial Sloan Kettering Cancer Center in New York City

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

MSKCC is an academic institution

What is your scientific area of research?

Adoptive Therapy of Cancer focused on the use of Genetically Modified Tumor Targeted T Cells

Why do you want to be a mentor?

Being aware of how important mentorship was to my career development and realizing the importance of experienced guidance for career development, I would very much like to honor my own mentors by providing similar mentor support to up and coming junior faculty.

As a mentor, what are you hoping to gain from this experience?

I feel that serving as a mentor to junior members of the society will not only help expand my abilities to serve as a mentor moving forward, but also give me the reward of feeling that I have potentially played a role in enhancing the careers of others. Given the rising significance of genetic approaches to treating disease, I feel that it is critical at this stage that the momentum be sustained. This momentum is perhaps best provided by the more junior hard working members of the society upon whom the future of our initial efforts are dependent.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 1
            b. Geographic Location - 3
            c. Professional Status / Success - 3
            d. Compatibility of Career Path - 2
            e. Communication Style - 4

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

As an MD, PhD student at SUNY at Buffalo, I worked in the laboratory of Stanley Spinola, MD gaining a PhD degree in microbiology with a strong emphasis on molecular biology. Having gained an interest, as a medical student, in oncology, I chose to pursue a fellowship in medical oncology at MSKCC after an internal medicine residency at Yale New Haven Hospital. Once at MSKCC I entered the laboratory of Michel Sadelain, MD, PhD where I began investigating a novel approach to the therapy of B cell cancers by introducing artificial T cell receptors, termed chimeric antigen receptors (CARs), targeted to the B cell antigen CD19 into patient T cells for subsequent reinfusion to the patients. This project has successfully been translated into the clinical setting wherein we now have 4 currently open protocols using this therapy to treat various B cell malignancies. Over my time at MSKCC I was uniquely able to become the PI of my own lab wherein we currently continue to investigate the biology of CAR modified T cells with a focus on the tumor microenvironment. We have additionally expanded our research program to include various other targeted antigens including those overexpressed on solid tumor malignancies.

Click here to view Renier Brentjens' Biosketch.

Jeffrey Chamberlain, PhD
University of Washington School of Medicine

What is your current professional status?

Professor of Neurology, Medicine and Biochemistry; McCaw Endowed Chair in Muscular Dystrophy, University of Washington School of Medicine, Seattle, WA.

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

University Medical Center

What is your scientific area of research?

Muscular dystrophy research, with a focus on genetics, gene therapy and myogenic stem cells.

Why do you want to be a mentor?

I enjoy working with students, post-docs and junior faculty and sharing my experiences. I think this is a good program for ASGCT, and I am eager to get involved. I think that the gene therapy field is maturing and it will be important to get more scientists involved and excited about the research.

As a mentor, what are you hoping to gain from this experience?

I hope to be able to encourage others in their research and to be able to contribute to faster progress in finding cures to human disease.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 3
            b. Geographic Location - 5
            c. Professional Status / Success - 4
            d. Compatibility of Career Path - 2
            e. Communication Style - 1
            f. Other: Enthusiasm - 1

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

My laboratory is developing therapeutics for the muscular dystrophies by performing both basic and translational research. Basic studies involve analysis of dystrophin structure and function, with a goal of better understanding the molecular pathways that contribute to muscular dystrophy. Our translational approach is focused on developing gene therapy using adeno-associated viral (AAV) vectors. Our structure/function studies led to the development of micro-dystrophin clones and the identification of methods for systemic delivery of genes to muscles of adult animals using AAV6. We are currently planning a phase 1 human trial using AAV/micro-dystrophin delivery. Finally, we are also exploring the isolation and transplantation of myogenic stem cells. Before moving to Seattle, I was a Professor of Human Genetics at Michigan and Director of the Center for Gene Therapy. I was an inaugural member of ASGCT and have been active in the Society since its founding.

Click here to view Jeffrey Chamberlain's Biosketch.

Stephanie Cherqui, Ph.D
University of California, San Diego

What is your current professional status?

Assistant Professor

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic institution

What is your scientific area of research?

Lysosomal storage disease, hematopoietic stem cells, lentiviral vectors, AAV, kidney targeted-gene delivery

Why do you want to be a mentor?

My mentors have been important in my careers and I still have great relationship with them. Thus, I want to give back and be able to advise young scientists who want to pursue a career in research.

As a mentor, what are you hoping to gain from this experience?

I am hoping to meet passionate young scientists and I am hoping that sharing my experience could help them in reaching their goal.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 3
            b. Geographic Location - 3
            c. Professional Status / Success - 4
            d. Compatibility of Career Path - 2
            e. Communication Style - 2
 

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

Dr. Cherqui received her Ph.D in 2002 from the University Rene Descartes (Paris, France), her research project focused on the lysosomal storage disorder cystinosis. Dr. Cherqui then specialized in stem cells and gene therapy during her post-doctoral internship at The Scripps Research Institute. In 2009, Dr. Cherqui was appointed as Assistant Professor at The Scripps Research Institute and started the project “Stem cells and gene therapy for cystinosis”. Using the mouse model for cystinosis, she showed that transplantation of hematopoietic stem cell progeny expressing a functional Ctns gene results in the significant improvement of the disease even if cystinosis is a progressive degeneration multiple tissue compartments and the protein involved a lysosomal transmembrane protein. A project focused on kidney-targeted gene delivery using AAV is also developed in the laboratory. In 2012, she joined the University of California San Diego as an Assistant Professor in the Department of Pediatrics, division of Genetics. The main focus of her laboratory is the clinical translation of her work on cystinosis and the fundamental understanding of tissue repair by bone marrow stem cells in the context of a non-hematopoietic genetic disease.

Click here to view Stephanie Cherqui's Biosketch.

Cynthia Dunbar, MD
NHLBI/NIH
Senior Principle Investigator, Section Head, Editor

What is your current professional status?

Senior Principle Investigator, Section Head, Editor

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Government Research Institution

What is your scientific area of research?

Hematopoiesis, Stem Cell Biology, Gene Therapy

Why do you want to be a mentor?

Well mostly because ASCGT asked me to do this, but I have been very committed to mentoring physicians and physician-scientists throughout my career, and I find the mentorship relationships the best part of my career.

As a mentor, what are you hoping to gain from this experience?

Continued satisfaction at career progression of mentees

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 2
            b. Geographic Location - 5
            c. Professional Status / Success - 3 
            d. Compatibility of Career Path - 1
            e. Communication Style - 3

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

Dr. Dunbar is a “triple threat”; with her entire career encompassing clinical investigation and patient care, basic science, and education/administration.  As a researcher, she has made important findings in stem cell biology, leukemogenesis and gene therapy, focusing on large animals to provide insights not possible using murine or in vitro models.  She has made important findings regarding the role of cytokines in leukemogenesis, functionally defined the frequency of stem cells in primates and provided numerous important insights into their behavior, and established herself as one of the leading investigators worldwide in hematopoietic stem cell gene therapy and insertional mutagenesis. She has also led important clinical trials in gene therapy, transplantation, autoimmune disease, and bone marrow failure. Her stature is evidenced by her invitations to chair and participate in key national and international meetings, serve on advisory panels and study sections, and a highly productive publication record. She graduated from Harvard College with a degree in History of Science, and from Harvard Medical School magna cum laude. Following internal medicine training at Boston City Hospital, she came to the NIH as a postdoctoral research fellow, and following one further year of clinical training in hematology at University of California, San Francisco, she returned to the NIH to set up her own research program. She was elected to the American Society for Clinical Investigation and the American College of Physicians. She has been active in major professional societies, including service on the executive committee of the American Society of Hematology, and was a founding member and three-time board member of the American Society for Cell and Gene Therapy. She served for ten years as an Associate Editor of the premier hematology journal BLOOD, and was named Editor-in-Chief in 2007, the first woman to serve in this position.  She has shown great commitment to education and career development of physician-scientists, running the NIH fellowship program for 17 years and serving as the chair of the NIH ACGME committee. She is a faculty member for an international training program in translational medicine, and on the board of the NIH Clinical Research Training Program. She has been a leader in efforts to improvement intramural NIH through her service on various committees and as a founding member and Co-Chair of the NIH Assembly of Scientists.  She has received numerous teaching and mentoring awards from NHLBI, NIH and her professional societies. She lives in Washington, DC with her husband and two daughters, and relaxes by performing and listening to classical music.

Cynthia Dunbar's CV coming soon.

Terence R. Flotte, MD
University of Massachusetts Medical School

What is your current professional status?

Celia and Isaac Haidak Professor of Medical Education
Dean, Provost and Executive Deputy Chancellor
Univeristy of Massachusetts Medical School

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

An academic institution

What is your scientific area of research?

Much was learned in the early cystic fibrosis clinical trials, including the paucity of AAV2 receptors on the luminal side of the airways. As a result, the lab recently designed a more robust expression cassette delivering a CFTR “mini gene” and has determined that AAV1 is superior to AAV2 and AAV5 at transducing airways. This new generation of viral vectors for CF is currently undergoing pre-clinical testing in preparation for human testing.

We are developing hybrid rAAV-RNAi approaches to treat AAT deficient liver disease. Given the need to down regulate Z-AAT, the lab has begun examining a number of innovative approaches to long-term expression of therapeutic RNAs using the rAAV platform.

Our lab is also studying the application of rAAV-based transduction of skeletal muscle for those Fatty Acid Oxidation Disorder deficiencies that are most common in humans. Through mouse models we are seeking to determine whether rAAV9 (vs. rAAV1) mediated transduction of skeletal and cardiac muscle will result in the improvement of acyl carnitine profiles and MRS profiles in mice with these disorders. This will lead to formal pre-clinical toxicology studies associated with muscle delivery of the targeted rAAV therapies in anticipation of new phase I clinical trials of gene therapy for these disorders.

Why do you want to be a mentor?

As a senior faculty member and medical school dean, I have spent much of my career mentoring medical and graduate students, fellows, and junior faculty. I find the mentoring process to be very enriching and rewarding. When successful, it allows one to see a greater cross-generational impact than would be possible if one focused solely on one’s own work.

As a mentor, what are you hoping to gain from this experience?

As pointed out above, I enjoy the interaction with young scientists at every stage of development. It is intellectually stimulating and personally rewarding, and allows one to feel that they are making a different that will endure well into the future, as the mentee (and ultimately their mentees) impact the advancement of science and medicine.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)           

            a. Compatibility of Scientific Specialties - 2
            b. Geographic Location - 3
            c. Professional Status / Success - 3
            d. Compatibility of Career Path - 2
            e. Communication Style - 2

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I am currently leading a research group that is investigating the delivery of therapeutic genes and microRNA with recombinant vectors for genetic diseases, such as alpha-1 antitrypsin (AAT) deficiency. In 1995, I was part of a team at Johns Hopkins that was the first to use rAAV, as a vehicle to deliver corrective genes to targeted sites in the body.  Since then, I have conducted several Phase 1 clinical trials for both AAT and Cystic Fibrosis as well as a Phase 2 clinical trial in AAT with vectors developed in my lab.

Click here to view Terry Flotte's CV.

Haiyan Fu, PhD
Nationwide Children's Hospital
Assistant Professor

What is your current professional status?

Assistant Professor

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic Instituion

What is your scientific area of research?

Gene Therapy, Neuological/Neurogenetic Diseases

Why do you want to be a mentor?

To help younger scientists for more efficient career development in the field, based on my own experience and lessons learnt.

As a mentor, what are you hoping to gain from this experience?

Broaden my communication with colleagues and learn the update development in the fiels.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 3
            b. Geographic Location - 5
            c. Professional Status / Success - 1
            d. Compatibility of Career Path - 2
            e. Communication Style - 1

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

The major focus of my research is effective CNS gene delivery for the treatment of neuropathic lysosomal storage diseases and neurological diseases in general.

Click here to view Haiyan Fu's CV.

Evanthia Galanis, M.D.
Mayo Clinic
Professor of Oncology

What is your current professional status?

Professor

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic Institution

What is your scientific area of research?

Oncolytic virotherapy in solid tumors, novel therapeutics for brain tumors

Why do you want to be a mentor?

To foster career development for junior investigators.

As a mentor, what are you hoping to gain from this experience?

The satisfaction of seeing talented young investigators launching successful careers and advancing the field; this is especially critical in order for viral and gene therapy approaches to become part of the standard of care options in cancer treatment.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 2
            b. Geographic Location - 5
            c. Professional Status / Success - 4
            d. Compatibility of Career Path - 1
            e. Communication Style - 3

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

Dr. Galanis is a Professor of Oncology and the Sandra J. Schulze Professor of Novel Therapeutics at the Mayo Clinic, where she also serves as the Chair of the Department of Molecular Medicine, and the Leader of the Gene and Virus Therapy Program of the Mayo Clinic Comprehensive Cancer Center.  Nationally she serves as an Advisor for the FDA’s Division of Biologics (CBER), she is a member of the Brain Tumor Steering Committee of the National Cancer Institute, and the Chair of the Neurooncology Committee of the Alliance for Clinical Trials in Oncology cooperative group.  Dr. Galanis’ laboratory effort focuses in the engineering and preclinical testing of oncolytic virus strains, viral retargeting and rational design of combinatorial strategies of oncolytic virotherapy with other treatment modalities such as radiation therapy and cell-cycle inhibitors.  In addition, she has been very active in implementing bench-to-bedside translation of novel oncolytic platforms, leading the translational effort which resulted in the first in-human testing of oncolytic measles virus strains in patients with ovarian cancer and glioblastoma multiforme as well as a recent translational effort, which culminated to first approved clinical trial in humans, using mesenchymal stem cells as vehicles to increase the efficiency of oncolytic virus delivery.  In addition, Dr. Galanis has been the principal investigator of multiple investigator-initiated clinical trials of viral therapeutics in solid tumors and cell cycle inhibitors/anti-angiogenesis agents in brain tumor treatment.

Click here to view Evanthia Galanis' CV.

Guangping Gao, PhD
University of Massachusetts Medical School

What is your current professional status?

Endowed full professor and Director of Gene Therapy Center at UMass Medical School

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic institution

What is your scientific area of research?

Gene therapy and miRNA function

Why do you want to be a mentor?

To strengthen the gene and cell therapy community by helping the professional growth of our next generation of researchers.

As a mentor, what are you hoping to gain from this experience?

To learn from young investigators and improve mentoring skills.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

a. Compatibility of Scientific Specialties - 1
b. Geographic Location - 3
c. Professional Status / Success - 2
d. Compatibility of Career Path - 4
e. Communication Style - 3

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

Dr. Guangping Gao received his Ph.D. in biological science in 1994 with his work involving the isolation and characterization of the human aspartoacylase gene and the genetic mutations responsible for Canavan disease, a severe form of inherited neurodegenerative diseases. He joined the University of Pennsylvania (UPenn) in 1994 where he has developed his career in viral vector biology for gene therapy. He made major contributions to the development of third generation adenovirus vectors and in 1996 was appointed as the Assistant Director of the Institute for Human Gene Therapy (IHGT) at UPenn with the primary responsibility of clinical adenovirus vector production. He was promoted to Associate Director and then to the Director of IHGT to oversee the vector discovery and development, process development, and vector core and quality control testing in 1998 and 2001 respectively. He was the driving force behind the discovery and vectorology of a novel primate adeno-associated virus (AAV) family and the development of simian adenovirus vector based vaccine programs.

Dr. Gao accepted a position at the University of Massachusetts Medical School (UMMS) in April 2008 as the Founding Director of the Gene Therapy Center and Vector Core, the Scientific Director of the UMMS-China Translational Research Initiative (which was later renamed UMMS-China Program), and Professor of Molecular Genetics and Microbiology. In September 2010, he was officially named the Penelope Booth Rockwell Chair in Biomedical Research, becoming one of the very few outstanding researchers at UMMS who receive named professorships.

Dr. Gao’s primary research interests include molecular mechanisms of AAV evolution and diversity, molecular interactions between endogenous AAV, vector and host genomes, molecular and cellular mechanisms of AAV vector transduction, micro RNA functions in adult mammals and gene therapy of Canavan Disease using rAAV to cross BBB for global CNS gene transfer and treating neuropathology and retinopathy.

He has published more than 120 papers in peer-reviewed journals and has 27 patented inventions. He is frequently invited to speak at international gene therapy conferences. He is among the investigators who are well recognized in the field of viral vector based gene therapy. He served on international committees such as the Membership Committee and International Committee of American Society of Gene and Cell Therapy, the AAV Vector Manufacturing and Reference Standard Committees, and Advisory Board of Advances in Experimental Medicine and Biology. He also served on the editorial boards of Human Gene Therapy and Gene Therapy. He is the senior Editor of Gene and Cell Therapy Book Series by the American Society of Gene and Cell Therapy and Springer Publisher.

Click here to view Guangping Gao's CV.

Paloma H. Giangrande, PhD
University of Iowa
*Junior Investigator*

What is your current professional status?

Assistant Professor

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic Institution

What is your scientific area of research?

RNA therapeutics

Why do you want to be a mentor?

I view mentoring as the most gratifying activity I perform on a daily basis.

As a mentor, what are you hoping to gain from this experience?

  Hoping to gain as mentor: To inspire mentees to identify their passion for research and pursue a career in scientific research.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

a. Compatibility of Scientific Specialties - 1
b. Geographic Location - 5
c. Professional Status / Success - 3
d. Compatibility of Career Path - 2
e. Communication Style - 3

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

Dr. Giangrande obtained her PhD in 1999 at Duke University in Pharmacology and Cancer Biology. In 2000, she joined the laboratory of Dr. Joseph Nevins where she worked on elucidating the differential roles of the E2F cell cycle regulators. After a four year postdoc, she joined the laboratory of Dr. Bruce Sullenger where she pioneered the use of RNA aptamers for targeted delivery of siRNAs. In 2007 Dr. Giangrande was appointed as Assistant Professor in the Dept. of Internal Medicine at the University of Iowa. Dr. Giangrande's research interests include developing RNA-based therapeutics to modulate cellular pathways underlying pathological cell proliferation in the setting of cancer and cardiovascular disease. A main interest is selecting RNA aptamers to receptors expressed on the surface of target-cells with SELEX (Systematic Evolution of Ligands by EXponential Enrichment) for the purpose of 1) modulating receptor function and/or 2) delivering therapeutic molecules (e.g. siRNAs, antimirs, drugs) into specific cell types.

Click here to view Paloma Giangrande's Biosketch.

Paola Grandi, PhD
University of Pittsburgh
Assistant Professor

What is your current professional status?

Assistant Professor

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic Institution

What is your scientific area of research?

Oncolytic vectors, brain tumors

Why do you want to be a mentor?

To develop scientists for the future

As a mentor, what are you hoping to gain from this experience?

The satisfaction to contribute to the next generation of scientists.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 1
            b. Geographic Location - 2
            c. Professional Status / Success - 3 
            d. Compatibility of Career Path - 3
            e. Communication Style - 3

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

 Paola Grandi, PhD, joined the Department of Neurological Surgery as an assistant professor in April of 2005. She has a joint appointment in the Department of Microbiology and Molecular Genetics. My research interests include studies to understand the development and progression of brain tumors emphasizing the role of miRNAs in cancer genetics.I have a long standing interest in the molecular biololgy of herpes simplex virus, mechanisms of virus replication and neuropathogenesis and virus host cells interactions that result in innate immune responses to infection.

Click here to view Paola Grandi's CV.

Uta Griesenbach, PhD
Imperial College Faculty of Medicine

What is your current professional status? 

Associate Professor/Reader in Molecular Medicine

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic Institution

What is your scientific area of research?

Gene and Cell Therapy for Lung Diseases

Why do you want to be a mentor?

I have a strong interest in teaching and have been involved in mentoring and tutoring students within our university for many years. This has always been a rewarding experience and I will enjoy supporting the ASGCT mentoring programme.

As a mentor, what are you hoping to gain from this experience?

The opportunity to provide others with encouragement and support, as well as experiencing the joy when mentees succeed in specific tasks will be very exciting and rewarding.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 3
            b. Geographic Location - 4
            c. Professional Status / Success - 5
            d. Compatibility of Career Path - 2
            e. Communication Style - 2

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I have been interested in cystic fibrosis (CF) gene therapy for almost 20 years and have very much enjoyed contributing to the highs and lows of lung gene transfer over the years. I have first worked in Toronto, Canada and for the last 16 years at Imperial College London. I have contributed to the development of viral and non-viral gene transfer agents and am particularly interested in the translational research.  I am a strategy group member in the UK CF Gene Therapy Consortium. In this role I am involved in pre-clinical and clinical research mainly related to lung gene therapy and biomarker development.

Click here to view Uta Griesenbach's Biosketch.

Scott O. Harper, PhD
Ohio State University & Nationwide Children's Hospital

What is your current professional status?

Associate Professor

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic Institution

What is your scientific area of research?

RNAi therapy; Muscular Dystrophy

Why do you want to be a mentor?

I benefitted from outstanding mentoring throughout my career, and I see it as an opportunity to pay it forward.  Also, I have many occasions to interact with trainees at my own institution and I think it will be fun and interesting to meet some from other locales.

As a mentor, what are you hoping to gain from this experience?

Broader perspective on issues affecting trainees nationwide that may apply to my own students and post-docs.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 4
            b. Geographic Location - 3
            c. Professional Status / Success - 2
            d. Compatibility of Career Path - 1
            e. Communication Style - 4

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

My name is Scott Harper.  I am currently an Assistant Professor of Pediatrics at The Ohio State University and a PI in the Center for Gene Therapy at Nationwide Children's Hospital in Columbus, Ohio.  My lab is focused on RNAi therapy for dominant myopathies, and we have a separate arm dedicated to more basic biological pursuits.  I did my grad school training at the University of Michigan from 1996-2002 (with a brief stop in Seattle at UW), followed by a post-doc at the University of Iowa from 2002-2007.  My wife and I had two children during my grad school years and a third when I was a post-doc, so I am familiar with the struggles of raising and uprooting a young family while pursuing a career in academic science.

Click here to view Scott Harper's CV.

Roland W. Herzog, PhD
University of Florida

What is your current professional status?

Professor fo Pediatrics, University of Florida

What is your work setting?

Academic Institution

What is your scientific area of research?

Hemophilia; AAV vectors; immunology

Why do you want to be a mentor?

I believe mentor and mentee can learn from each other. Also, lasting connections and relationships with other scientists help drive the field forward.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 1
            b. Geographic Location - 4
            c. Professional Status / Success - 2
            d. Compatibility of Career Path - 2
            e. Communication Style - 3

Biography:

Roland W. Herzog, Ph.D. is Professor of Pediatrics, Molecular Genetics and Microbiology at the University of Florida. He is member of the Division of Molecular and Cellular Therapy and the Powell Gene Therapy Center. After completing a PhD in Microbiology (Auburn University, 1996) and postdoctoral training (Children’s Hospital of Philadelphia 1996-2000), Dr. Herzog became assistant professor at the University of Pennsylvania, before joining UF in 2005. He received a Career Development Award from the National Hemophilia Foundation, an Outstanding New Investigator Award from ASGCT, a Bayer Hemophilia Award, and a Basic Science Award from the UF College of Medicine. Dr. Herzog serves on the editorial boards of Current Gene Therapy and Human Gene Therapy, as Deputy Editor of Molecular Therapy, and as Executive Editor of the Journal of Genetic Syndromes & Gene Therapy. He is member of the NIH study section Hemostasis and Thrombosis. Dr. Herzog has published two textbooks and >100 articles, and has received multiple federal grants for his research program.

Click here to view Roland Herzog's Biosketch.

Mark A. Kay, MD, PhD
Stanford University School of Medicine

What is your current professional status?

Professor of Pediatrics and Genetics, Stanford University

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic

What is your scientific area of research?

Gene therapy and non coding RNA biology

Why do you want to be a mentor?

I would like to give back what was given to me and provide leadership, which I hope will help talented new investigators to reach their full potential.

As a mentor, what are you hoping to gain from this experience?

Watching skilled new investigators mature and prosper.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 1
            b. Geographic Location - 3
            c. Professional Status / Success - 1
            d. Compatibility of Career Path - 3
            e. Communication Style - 2

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

Dr. Kay is the Dennis Farrey Family Professor in the Departments of Pediatrics and Genetics at Stanford University. He is head of the Division of Gene Therapy and serves as the Vice Chair for Basic Research in Pediatrics. Dr. Kay is clinically trained, and previously certified in pediatrics, medical & biochemical genetics,

Dr. Kay’s group has published ~200 papers with 30 in the highest profile journals. He has been a true bench-to-bedside physician-scientist with the first demonstration of therapeutic rAAV transduction of liver in both small and large animal models, working out the molecular process of AAV transduction in vivo, and then playing a major role in a clinical trial representing the first systemic delivery of rAAV in humans. He has uncovered important principles involved in plasmid DNA vectors and developed a robust means to produce minicircle DNA vectors which remain episomal yet provide sustained transgene expression in quiescent tissues. These vectors also shows promise in the creation of ips stem cells. He has led the field in demonstrating RNAi activity in mammals and against human viral infections in mammals. His work along the way has uncovered the mechanisms of how miRNAs regulate genes as well as a new small RNAs and molecular processes, which have not been previously identified in vertebrates. He is recognized as an international leader based on his activities activities, awards and service including the Mead Johnson Researcher of the Year, and past president of the American Society of Gene Therapy of which he was a founding member. He has a tremendous training record spawning 17 academic faculty (>1/3 of his total trainees).

Click here to view Mark Kay's CV.

Hans-Peter Kiem, MD, FACP
Fred Hutchinson Cancer Research Center

What is your current professional status?

José Carreras/E. Donnall Thomas Endowed Chair for Cancer Research
Associate Head and Member, Fred Hutchinson Cancer Research Center

Professor of Medicine / Adjunct Professor of Pathology
University of Washington School of Medicine

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic Institution:
Fred Hutchinson Cancer Research Center and University of Washington
1100 Fairview Avenue North
Seattle, WA 98109

What is your scientific area of research?

The main focus of the my lab is to study stem cell biology and stem cell gene transfer with the goal of developing novel stem cell-based treatment strategies for patients with genetic, infectious stem cells (HSCs) although more recently we have also initiated studies using embryonic stem (ES) cells and induced pluripotent stem (iPS) cells. Hematopoietic stem cells are attractive targets for gene therapy because of their ability to permanently reconstitute the hematopoietic and immune systems. However, in order to efficiently modify HSCs one has to be able to identify and isolate HSCs and identify vector systems that allow for efficient transduction of HSCs. In recent studies we have demonstrated efficient gene transfer with lentivirus and foamy virus based vector systems. We have shown successful expansion and in vivo selection of hematopoietic repopulating cells. Based on these studies we have currently an open gene therapy study using chemoprotected stem cells for the treatment of patients with glioblastoma and we have a clinical gene therapy study to correct the genetic defect the marrow of patients with Fanconi anemia. We are also working on studies for the correction of other genetic diseases using lentivirus vectors and gene repair strategies. We are exploring anti-HIV stem cell gene therapy strategies using lentivirus and foamy virus vectors and zinc finger technology. In addition to the hematopoietic stem cell work we are pursuing the generation of iPS cells from large animal models to study iPSC-based treatment approaches. We are also exploring novel cancer treatment strategies using oncolytic viruses.

Thus current research in our laboratory focuses on studies to:

• characterize HSCs from large animal models
• investigate hematopoietic differentiation of iPSCs
• analyze the integration site pattern of different retroviruses
• analyze the clonal composition of hematopoiesis after transplantation of gene-modified HSCs
• develop preclinical protocols using stem cell gene therapy to facilitate allogeneic nonmyeloablative stem cell transplantation
• develop clinical gene therapy protocols for genetic and acquired diseases including cancer
• study the generation of induced pluripotent stem cells from animal models
• study the use of oncolytic gene therapy approaches for cancer

Why do you want to be a mentor?

As part of my job as a physician scientist I have mentored many students and fellows in the lab and clinical setting and have always enjoyed the teaching and mentoring aspects of my work. I think it is crucial for the development of young scientists to have good and engaged mentors.

As a mentor, what are you hoping to gain from this experience?

To help young investigators be successful in their academic careers. To be connected with young investigators and learn about issues and problems and hopefully find ways to help.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)            

            a. Compatibility of Scientific Specialties - 2
            b. Geographic Location - 5
            c. Professional Status / Success - 5
            d. Compatibility of Career Path - 2
            e. Communication Style - 3

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I am a physician scientist in the field of gene therapy and transplantation. I am interested in stem cell biology and how we can apply gene and stem cell therapies to treat different diseases like genetic and infectious diseases and cancer. My lab has studied stem cell biology and gene therapy strategies in large animal models and we have translated our findings to the clinic and currently have 2 open clinical gene therapy studies. My lab is also exploring novel ways of generating induced pluripotent stem cells and expanding hematopoietic stem cells. We are also developing novel tools for gene targeting approaches for the treatment of genetic diseases and HIV/AIDS.

Click here to view Hans-Peter Kiem's CV.

Karen Kozarsky, PhD
ReGenX Biosciences, LLC

What is your current professional status?

Vice President, Research & Development at ReGenX Biosciences (a gene therapy company).

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Small biotech company

What is your scientific area of research?

Gene therapy for inherited and acquired diseases using adeno-associated virus (AAV).

Why do you want to be a mentor?

I would like to be able to contribute to other people’s growth and success. People have mentored me in both big and small ways, and I would like to return the favor by doing the same for others.

As a mentor, what are you hoping to gain from this experience?

I would like to add to my community within the gene therapy field, and to share my experience with others.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 1
            b. Geographic Location - 5
            c. Professional Status / Success - 4
            d. Compatibility of Career Path - 3
            e. Communication Style - 2

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I have been in the gene therapy field for 20 years, in research staff and then junior faculty positions at the University of Michigan and subsequently at the University of Pennsylvania. I then moved to GlaxoSmithKline (formerly SmithKline Beecham), where I headed their gene therapy effort. My earliest work was developing the use of recombinant adenovirus, focusing on cholesterol metabolism, and moved into the development and characterization of novel AAV vectors through a sponsored research agreement with Jim Wilson’s lab at Penn. I am working on therapeutics based on this AAV platform for the eye, liver/metabolic disorders, and CNS applications.

Click here to view Karen Kozarsky's Biosketch.

Pedro Lowenstein, MD, PhD
University of Michigan School of Medicine

What is your current professional status?

Professor of Neurosurgery and Cell and Developmental Biology, University of Michigan Medical School

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

The University of Michigan: public academic institution

What is your scientific area of research?

Neuro-Oncology, Immunology, Immunotherapies for the treatment of cancer, Mathematical modeling

Why do you want to be a mentor?

I like teaching the younger generation. I have worked in many different countries on three different continents. I have, thus, a wide understanding of the challenges of doing science all over the world. Also, as an MD and PhD engaged in translational medicine, I understand the challenges to take a new idea all the way into a clinical trial. I very much enjoy sharing my enthusiasm and experience, and help the younger generations to find their own ways. I like to provide support at a stage when strong support can make or break a budding career.

As a mentor, what are you hoping to gain from this experience?

Share my experience and help the next generation move even further. The pleasure of seeing young people supported in their dreams.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 1
            b. Geographic Location - 3
            c. Professional Status / Success - 4
            d. Compatibility of Career Path - 2
            e. Communication Style - 2
            f. Other - Passion for the adventure of science

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I am currently a Professor of Neurosurgery at the University of Michigan. I am interested in finding new ways to treat deadly brain tumors. We must first understand the biology of brain tumors to identify their Achilles tendons. In spite of much work over the last 70 years, the prognosis in brain tumors has improved very little. My team is now working towards understanding how brain tumors grow, disperse, and eventually kill patients. Using a wide array of techniques spanning form cell and molecular biology all the way to stem cells, system biology, and more recently mathematical modeling, we are discovering how tumors actually grow silently within the confined space of the brain, how they interact with the brain, and why they eventually kill patients. The new approaches are providing unexpected ways to treat brain tumors, which we are converting into gene therapy clinical trials.

Click here to view Pedro Lowenstein's Biosketch.

R. Scott McIvor, PhD
University of Minnesota

What is your current professional status?

Professor of Genetics, Cell Biology and Development
Director, Gene Therapy Program
Institute of Human Genetics
University of Minnesota, Minneapolis, Minnesota

Chief Executive Officer, Chief Science Officer
Discovery Genomics, Inc.
Minneapolis, Minnesota, 55413

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic Institution / Small Biotech Startup

What is your scientific area of research?

Gene Therapy for genetic disease and cancer

Why do you want to be a mentor?

To assist young investigators in forging a pathway to success, and thus contribute to development of the gene therapy field.

As a mentor, what are you hoping to gain from this experience?

Sense of contribution to the field and a chance to interact with active young scientists in the field.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 3
            b. Geographic Location - 4
            c. Professional Status / Success - 3
            d. Compatibility of Career Path - 2 
            e. Communication Style - 2

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

My background includes nearly 30 years experience in the gene therapy field, studying both viral and non-viral (Sleeping Beauty) gene transfer systems. As a postdoctoral fellow I conducted some of the earliest work on use of retroviral vectors for therapeutic gene transfer in hematopoietic stem cells. With two tours of duty on the NIH Recombinant DNA Advisory Committee and 12 years of NIH study section service, I have extensive experience and familiarity with gene therapy preclinical and clinical study design. Current research projects include AAV and lentiviral vectors for therapy of lysosomal storage diseases emphasizing neurologic outcomes, lentiviral vectors for therapy of radiation-sensitive SCID, Sleeping Beauty (SB) vectors for hemophilia, and SB vectors for therapy of colorectal cancer. As a part of my research program at the University of Minnesota, I have trained 13 graduate students and 4 postdoctoral fellows. I have participated in the evaluation of faculty mentoring programs at the University of Minnesota, and in my teaching I am particularly committed to conveying key elements of research proposal writing and submission to achieve successful funding.

 Click her e to view  R. Scott McIvor's CV.

Andra Miller, PhD
Biologics Consulting Group
Director

What is your current professional status?

Director, Cell and Gene Therapies

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Consulting Group/Home Office

What is your scientific area of research?

Molecular Genetics/Developmental Biology

Why do you want to be a mentor?

Interested in helping people interested in a career in regulatory

As a mentor, what are you hoping to gain from this experience?

Meet people with similar interests and supporting them in their career choices

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 2
            b. Geographic Location - 3
            c. Professional Status / Success - 4
            d. Compatibility of Career Path - 2
            e. Communication Style - 2

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I provide guidance and regulatory strategies to scientists, clinicians and industry to facilitate the development of cell and gene based therapies leading to clinical trails in indications ranging from cancer to diabetes, heart disease, muscular disorders and infectious disease. Because of this I have had the opportunity to work with many of the gene therapy systems currently in use, including integrating and non-integrating, viral and non-viral vector systems. In this capacity I interface with academia, industry and the regulatory agencies.  In addition, my expertise includes interpretation of FDA and NIH/RAC regulations/guidelines, Phase I, II and III product development strategies, FDA submissions, and assessment of cGMP, cGTP and quality system compliance.

Click here to view Andra Miller's CV.

Ko Mitani, PhD
Saitama Medical University
Professor

What is your current professional status?

Professor of Gene Therapy Division, Research Center for Genomic Medicine

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic Institution

What is your scientific area of research?

Gene repair therapy targeting human stem cells using viral vectors

Why do you want to be a mentor?

I would like to help young scientists to explore their potential and develop their career in the gene therapy field.

As a mentor, what are you hoping to gain from this experience?

I hope to have great and stimulus discussion with young scientists, who have unlimited potential and more flexible brain than me!

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 3
            b. Geographic Location - 5
            c. Professional Status / Success - 3 
            d. Compatibility of Career Path - 3
            e. Communication Style - 1

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

After obtaining the Ph.D. degree at the Univ. of Tokyo, I received post-doctoral training in Dr. Caskey’s lab at Baylor College of Medicine from 1990 to 1993, where we developed the helper-dependent gutless adenoviral vector system.  After spending two years at the Univ of Tokyo as a Lecturer, I became an Assistant Professor at UCLA School of Medicine, where my lab developed various hybrid adenoviral vectors for stable gene transfer.  After coming back to Japan in 2003, our efforts have been towards gene repair therapy for inherited disorders. In combination with recent development of iPSC technology, we showed that gene targeting using adenoviral vector is extremely efficient in human ESCs/iPSCs.  We have been improving the technology for both chromosomal manipulation and repair of disease-causing mutations in human stem cells.  My experience on both sides of the Pacific Ocean would hopefully help young international scientists to enhance their career.

Click here to view Ko Mitani's CV.

Eirini P. Papapetrou, MD, PhD
University of Washington
*Junior Investigator*

What is your current professional status?

Assistant Professor at the University of Washington

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic institution

What is your scientific area of research?

Human iPS cells for modeling and gene therapy of blood diseases

Why do you want to be a mentor?

As a very recent junior faculty, I appreciated the importance of advice from colleagues who recently went through the process of transitioning from post-doc to independent investigator, and so would like to share my experience on finding an academic job and setting up a new laboratory with junior scientists who are at this stage of their career.

As a mentor, what are you hoping to gain from this experience?

To help junior scientists find a fulfilling career path and navigate the academic job search.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 4
            b. Geographic Location - 2 (USA)
            c. Professional Status / Success - 2
            d. Compatibility of Career Path - 1 (students or post-docs who aspire to a career in academia)
            e. Communication Style - 3

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I obtained an MD and PhD in Greece and trained in internal medicine and hematology. In 2006 I moved to New York as a post-doctoral fellow in Michel Sadelain’s laboratory at Memorial Sloan-Kettering Cancer Center. My post-doctoral research focused initially on lentiviral gene transfer into hematopoietic stem cells and miRNA regulation and subsequently on the generation and genetic modification of human induced pluripotent stem (iPS) cells. In January 2012 I started my own laboratory at the University of Washington in Seattle. My lab uses human pluripotent stem cells, mainly iPS cells, to study normal and abnormal hematopoiesis and develop new technologies towards translation of iPS cells to the clinic. We are using reprogramming of patient-derived hematopoietic cells and genetic engineering of normal human pluripotent stem cells to develop new models of dysplastic hematopoiesis and bone marrow failure. We are also developing new approaches to generate safer genetically modified iPS cells for cell therapies.

Click here to view Eirini Papapetrou's CV.

Matthew M. Porteus, MD, PhD
Stanford University School of Medicine
*Junior Investigator*

What is your current professional status?

Associate Professor of Pediatrics, Stanford University

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.):

Academic Institution

What is your scientific area of research?

Homologous Recombination Based Gene Therapy, Transplantation of Hematopoietic Stem Cells

Why do you want to be a mentor?

It is rewarding and a chance to “pay it forward” for the mentoring I have received in my career.

As a mentor, what are you hoping to gain from this experience?

I expect that I will learn a substantial amount and will help me broaden my expertise.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 1
            b. Geographic Location - 3
            c. Professional Status / Success - 3
            d. Compatibility of Career Path - 1
            e. Communication Style - 1

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I am a physician-scientist trained in pediatric hematology-oncology with a longstanding interest in developing homologous recombination based approaches to gene therapy, particularly genetic diseases of the blood. I did my clinical training at Boston Children’s Hospital/Dana Farber Cancer Institute and my post-doctoral research training with Dr. David Baltimore at MIT and then CalTech. I was an assistant professor in Pediatrics and Biochemistry at UT Southwestern from 2/2003-9/2010 and have been an associate professor of Pediatrics at Stanford since 10/2010.

Click here to view Matthew Porteus' CV.

Rachel Salzman, DVM
The Stop ALD Foundation

What is your current professional status?

Chief Scientific Officer and Industry Advisor

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Not for Profit Foundation and private sector

What is your scientific area of research?

Advancing gene and cell therapy in rare disease

Why do you want to be a mentor?

I am interested in helping investigators better navigate their profession so that they can make career choices that will result in their work ultimately be translatable towards benefiting patients. To be successful in this goal one must be aware and proactive when it comes to identifying appropriate partnerships and opportunities that exist within the scientific and patient advocacy communities along with understanding industry's role in this narrative. I believe that a mentee will gain a higher level of job and life satisfaction as a result of developing productive relationships which will allow for optimal leveraging of biomedical research and knowledge.

As a mentor, what are you hoping to gain from this experience?

As someone who works in "ultra rare" disease I am acutely aware of the power and importance of the individual. I would greatly value being able to impact and optimize even one person's career path as I see this directly benefiting future patients and their loved ones.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 3
            b. Geographic Location - 5
            c. Professional Status / Success - 2
            d. Compatibility of Career Path - 2
            e. Communication Style - 4

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

Dr. Salzman serves as Chief Scientific Officer of The Stop ALD Foundation  (SALD), and has held this position since 2001.  SALD is a non-profit Medical Research Organization dedicated to employing entrepreneurial approaches and innovative methodology towards effective therapies, cures, and prevention of x-linked adrenoleukodystrophy (ALD), an often fatal neurodegenerative disease.  The biomedical interests of the Foundation include gene therapy, hematopoietic stem cells, mesenchymal and other adult stem cells, genomics, and small molecules.  In this context SALD made critical contributions to the groundbreaking ex vivo lenti trial conducted in France.  Rachel also consults for large pharma and biotech in the field of drug development, including preclinical and clinical analysis in a variety of therapeutic areas including oncology, metabolic disease, CNS disorders, and orphan indications.  Prior to these roles, Rachel worked in private veterinary practice in both large and small animal medicine.  She has a D.V.M. from Oklahoma State University and a B.S. from Rutgers University.

Rachel Salzman's CV coming soon

Andrew M. Scharenberg, MD
Seattle Children's Research Institute

What is your current professional status?

Professor, Departments of Pediatrics and Adjunct Professor, Department of Immunology, University of Washington and Seattle Children’s Research Institute, Seattle, WA

Chief Scientific Officer, Cellectis Therapeutics, Paris, France

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

In my academic work, I am affiliated with two academic institutions, the University of Washington and Seattle Children’s Research Institute. I run a 10 member laboratory and attend at Seattle Children’s Hospital on the Immunodeficiency Service.

In my role as Chief Science Officer at Cellectis Therapeutics, I am charged with setting the overall strategy for therapeutics development. This role presently involves managing a 16 person group involved in projects ranging from technology development to clinical process scale up.

What is your scientific area of research?

Cell engineering with rare cleaving nucleases.

Why do you want to be a mentor?

As a physician who has dedicated his career to developing cell and gene therapies, I am interested in seeing them make an impact on the practice of medicine. In my view, there is no better way to do this than by helping talented people work in the field. Serving as a mentor provides a venue for helping someone entering cell and gene therapy identify the best opportunities available for their particular background and talents. In addition, building a career in an emerging field, such as cell and gene therapy, involves navigating a variety of challenges. My combined background in both academic and commercial realms of cell and gene therapy has required me to navigate a diverse set of such challenges. Thus, I can offer a very broad perspective on how to develop an interesting and rewarding career, while hopefully avoiding some of the frustrations inherent in working at the cutting edge of technology.

As a mentor, what are you hoping to gain from this experience?

Being a mentor is a great opportunity to get to know a talented scientist I might not otherwise have a chance to meet, and to learn something new from them. I find that I never fail to be inspired by discussions with other interested and motivated investigators, who share a mutual interest in science, and a desire to translate their science into an impact on people (patients!) and society.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

1. Compatibility of Scientific Specialties  (3 – I think some overlap in interests is important, but does not need to be complete).
2. Geographic Location (2 – probably helps to be able to meet in person at least semi-regularly)
3. Professional Status / Success (2 – I think it would be hard to effectively mentor someone who I did not perceive as highly motivated to succeed).
4. Communication Style (3/4 – I think a variety of communication styles can be effective, and part of being a mentor is providing feedback on exactly this type of issue).

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I am an attending physician at Seattle Children’s Hospital; Professor, Department of Pediatrics; and Adjunct Professor, Department of Immunology, University of Washington. At Seattle Children’s Hospital, I serve as attending physician in the Immunodeficiency Clinic and on the inpatient Immunodeficiency service, and provide interpretative services on B-cell immunophenotyping for the Immunology Diagnostic Laboratory. My laboratory at Seattle Children’s Research Institute focuses on the development of technology for genome engineering, with a translational focus on hematopoietic stem cells and T-cells. I also serve as co-director of the Northwest Genome Engineering Consortium, a group of interdisciplinary investigators in the Seattle area working on applications of genome engineering technology for the treatment of inherited diseases and cancer. I am a co-founder of Pregenen, for which I presently serve as a member of the Board of Directors. I also presently serve as Chief Scientific Officer for Cellectis Therapeutics, where I provide strategic direction for therapeutic applications of Cellectis nuclease platforms and manage the Ctx therapeutic team.

Click here to view Andrew Scharenberg's Biosketch.

Jackie Sheng, PhD
Amgen, Inc
Research Director

What is your current professional status?

Research Director

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Bio-industry/Pharmaceutical Company

What is your scientific area of research?

Very broad, mainly focus on drug development

Why do you want to be a mentor?

I desire to share my experiences of working for a bio-industry and my knowledges of drug development to help young scientits on their choice of career paths.

As a mentor, what are you hoping to gain from this experience?

Satisfactory from helping young scientists and also networking.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 3
            b. Geographic Location - 3
            c. Professional Status / Success - 4
            d. Compatibility of Career Path - 3
            e. Communication Style - 2

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I am a research director at Amgen. I earned my Ph.D. in Molecular and Biochemistry from University of Miami and subsequently did my postdoc training from Purdue University and UT Southwestern Medical School. I have since been working for Amgen with responsibilities of many different areas such as target validations using viral vectors, RNAi, transgenic and knockout and next generation sequencing , as well as therapeutic protein development.  My current responsibility is to overseen a group of thirty scientists to support early drug discovery and development. Amgen is modality independent and seeks opportunities in many areas including gene and cell therapies. I have been one of the main scientists help the company on due diligence and evaluating the technologies.

Click here to view Jackie Sheng's CV

Sonia I. Skarlatos, PhD
NHLBI
Deputy Director

What is your current professional status?

Deputy Director, Division of Cardiovascular Diseases at NHLBI

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Government Organization

What is your scientific area of research?

Oversee programs in gene and cell therapy for cardiovascular diseases

Why do you want to be a mentor?

I can help answer questions about grant mechanisms and grant process at the NIH and NHLBI

As a mentor, what are you hoping to gain from this experience?

Learn what junior investigators understand about the grant process

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 4
            b. Geographic Location - 5
            c. Professional Status / Success - 4
            d. Compatibility of Career Path - 4
            e. Communication Style - 4

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I am responsible for the overall planning, development and implementation of national and international research activities for heart and vascular diseases, and the Program Director of three NHLBI programs.

Click here to view Sonia Skarlatos' CV.

Arun Srivastava, PhD
University of Florida College of Medicine

What is your current professional status?

George H. Kitzman Professor of Genetics and Chief
Division of Cellular and Molecular Therapy
Departments of Pediatrics, Molecular Genetics & Microbiology
Powell Gene Therapy Center

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.) 

Academic Institution (University of Florida)

What is your scientific area of research?

Development of AAV vectors for human gene therapy, and AAV-host cell interactions.

Why do you want to be a mentor?

To share my experience with the younger generation of scientists.

As a mentor, what are you hoping to gain from this experience?

To learn from the younger generation of scientists.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 1
            b. Geographic Location - 5
            c. Professional Status / Success - 5
            d. Compatibility of Career Path - 3
            e. Communication Style - 5

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I received my PhD degree from the Indian Institute of Science in Bangalore, India. After completing my postdoctoral training at the Memorial Sloan-Kettering Cancer Center in New York, I worked as a Research Associate at the University of Florida. For nearly two decades, I was on the faculty at Indiana University School of Medicine in Indianapolis, where I rose to the rank of Professor. I was recruited back to the University of Florida in 2004. For the past 30 years, my research has been focused on the following two parvoviruses, the non-pathogenic adeno-associated virus (AAV), and a common human pathogen, the parvovirus B19, and the development of recombinant parvovirus vectors for human gene therapy. The current emphasis is on developing recombinant vectors for gene therapy for genetic diseases such as thalassemia and sickle cell disease, and malignant disorders such as hepatoblastoma and hepatocellular carcinoma. In the past three decades, I have mentored 8 Clinical Fellows and 32 Postdoctoral Fellows. Two students have graduated with MS degrees, and 7 students have received their PhD degrees from my laboratory. My research activities are currently supported by grants from the National Institutes of Health and the Bayer Hemophilia Program. I have also been awarded 3 US Patents on my research on human parvoviruses and their potential use as vectors in human gene therapy. I currently serve on an NIH Study Section as well as on the Editorial Boards of Journal of Virology, Human Gene Therapy, Recent Patents on DNA and Gene Sequences, Gene Therapy and Molecular Biology, and Journal of Chinese Integrative Medicine. I also serve as an Executive Editor of the Journal of Genetic Syndromes and Gene Therapy. I have published 157 research articles, mostly on human parvoviruses, in peer-reviewed journals, reviews, and book chapters.

Click here to view Arun Srivastava's CV.

Renata M. Stripecke, PhD
Hannover Medical School

What is your current professional status?

Associate Professor

Head of the Lymphatic Cell Therapy Laboratory from the Excellence Cluster REBIRTH

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic Institution

What is your scientific area of research?

 Hematology, Cancer, Chronic Infections, Immunology and Gene Therapy. The focus in my research group is on dendritic cells (DC), which have a pivotal role for immunologic surveillance against cancer and chronic infections.

Why do you want to be a mentor?

I moved to Germany in 2007 and we were able to establish a nice team. I have supervised post-docs, pre-docs, master students and medical students and I enjoy doing it.  My teaching activities in our international PhD programs have been devoted to immunology (“Antigen Presenting Cells”) and molecular biology/ gene expression (“Recombinant Vector Systems”), which are also main topics of my scientific training.  Thus, I feel that I can contribute to “new minds” growing in science. In addition, scientific careers are sometimes not “linear”, and everyone experience some bumps here and there. It was very important for me to have mentors to give me energy and perspectives for focusing on what was important to reach my academic goals.

As a mentor, what are you hoping to gain from this experience?

Internationalization, scientific mobility, career/family balance and career development for women are important academic topics for me, which I support and would like to further develop in our Medical School in Germany.  It is therefore great for me to keep in touch with the new academic generations in the US, since so much is done there regarding these topics.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 1
            b. Geographic Location - 4
            c. Professional Status / Success - 3
            d. Compatibility of Career Path - 3
            e. Communication Style - 2

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I am quite cosmopolitan, as I was born and grew up in Brazil, where I got my BS and MS in Genetics (UNICAMP), then moved to Germany for my PhD in molecular biology (EMBL), then to the US for postdoctoral training and assistant professor positions in gene and immune therapy (USC, UCLA), and finally as an associate W2 professor to Germany (MHH). My expertise is on reprogramming the immune system using recombinant vectors in order to create potent vaccines to be used to prevent, control or eradicate chronic diseases. Due to their central role in orchestration of cellular and humoral responses, DC are a main cell target for development of immune therapeutic products and vaccines. Through molecular approaches, we are devising ways to genetically reprogram DC precursors to differentiate into multipotent induced DC. Our experience with lentiviral vectors has resulted in rational vector designs for generation of lentiviral-induced dendritic cells (“iDC”). Over the past years, we demonstrated the feasibility of multicistronic integrase-defective vectors expressing combinations of cytokines and antigens to enable the differentiation of DC designed to different cancer types and chronic infections. It was thus possible to demonstrate that this new technology is modular and clinically adaptable to GMP production for use in immune regeneration against cancer or viral infections. Allied with these translational goals, we are exploring iDC as a tool to improve humanization of immune deficient mice. In this context, we are also exploring the use of iDCs to regenerate immune responses in lymph nodes.

Click here to view Renata Stripecke's CV.

Bakhos A. Tannous, PhD
Massachusetts General Hospital & Harvard Medical School
*Junior Investigator*

What is your current professional status?

Associate Professor and Principle Investigator at the Massachusetts General Hospital and Harvard Medical School

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic Institution

What is your scientific area of research?

Brain cancer, Gene/cell/drug therapy

Why do you want to be a mentor?

To help young investigators in advancing their career.

As a mentor, what are you hoping to gain from this experience?

Mentoring experience

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 2
            b. Geographic Location - 3
            c. Professional Status / Success - 3
            d. Compatibility of Career Path - 3
            e. Communication Style - 1

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I received my Ph.D. from the University of Windsor, Canada in Clinical Chemistry followed by post-doctoral training at the Center for Molecular Imaging Research and Neuroscience Center at the Massachusetts General Hospital (MGH) and Harvard Medical School (HMS). Currently, I am an Associate Professor at HMS and Associate Neuroscientist at MGH. My research focuses on developing novel imaging and gene/cell therapeutic strategies against brain tumors with three main focus: (1) developing molecular biosensors which reports from tumors environment; (2) targeting brain tumors using different gene transfer technologies and neural stem cells; (3) high throughput screening for drugs in combination with gene/cell therapy which acts specifically against brain tumor stem-like cells.

Click here to view Bakhos Tannous' Biosketch.

Luk H. Vandenberghe, PhD
Schepens Eye Research Institute & Massachusettes Eye and Ear Infirmaru, Harvard Medical School

What is your current professional status?

Principal Investigator, Massachusetts Eye and Ear Infirmary, Harvard Medical School

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic

What is your scientific area of research?

Vector development, Ocular Gene Therapy

Why do you want to be a mentor?

 I chose to be a mentor to pass on my experiences and guide new investigators to an independent and successful career. New blood and creativity will ultimately strengthen gene therapy as a field.

As a mentor, what are you hoping to gain from this experience?

 I hope to meet passionate people with fresh ideas

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 2
            b. Geographic Location - 4
            c. Professional Status / Success - 3
            d. Compatibility of Career Path - 3
            e. Communication Style - 1

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

My laboratory focuses on building our gene therapy as a platform therapeutic in ophthalmology. We therefore identify, study and aim to overcome translational hurdles for this to become reality. Briefly, we are working on novel vector development, immunology of ocular gene transfer, animal modeling, and translation of several retinal gene transfer approaches. We believe that scientific efforts in this ambitious field need to go hand in hand with active research of the policy and economical aspects of our ambition to bring gene therapy to the clinic.

Click here to view Luk Vandenberghe's Biosketch.

Zejing Wang, MD PhD
Fred Hutchinson Cancer Research Center and University of Washington

What is your current professional status?

AssistantProfessor

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Nonprofit Research Institute

What is your scientific area of research?

Gene Therapy, Muscular Dystrophies, Immunology and Immune Modulation, Translational Reserach

Why do you want to be a mentor?

Realizing the importance of guidance and support of a mentor and mentors in one’s career development, hope to share my experience of what worked and did not work for me in my career development, and to provide support and guidance to junior members of the society in finding their career path.

As a mentor, what are you hoping to gain from this experience?

This is not only an opportunity to make contribution to the society, but the experience will also expand my skills, knowledge and abilities to serve as a mentor.  It will also give me the reward of feeling when making positive impact on enhancing others’ career development.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 2
            b. Geographic Location - 4
            c. Professional Status / Success - 3
            d. Compatibility of Career Path - 2
            e. Communication Style - 4

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

I am a Research Assistant Professor of Medicine at University of Washington and Associate in Clinical Research at the Fred Hutchinson Cancer Research Center.  My current research focuses are on preclinical translational research in large animal model, developing strategies for efficient adeno-associated viral vector (AAV)-mediated gene therapy for treating muscular dystrophies.  Current projects focus on studies of the efficacy and safety of intramuscular and intravascular delivery of vectors to skeletal and cardiac muscles.  Projects also include studying of host immune responses to virus and immune modulation for overcome immune barriers. These studies form the basis of a phase I human clinical trial in planning using AAV for gene delivery to muscles of dystrophy patients.

Click here to view Zejing Wang's CV.

Andy Wilber, PhD
Southern Illinois University School of Medicine
*Junior Investigator*

What is your current professional status?

Assistant Professor

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic Institution

What is your scientific area of research?

Hematopoietic Stem Cell Gene Transfer for Severe Hemoglobin Disorders and Anti-tumor Immunity

Why do you want to be a mentor?

Having recently transitioned from a post-doctoral fellow to Assistant Professor, I hope to use my experience to help associate members of the society do the same. I hope to assist in the development of a CV, biosketch, and research plan toward this goal.

As a mentor, what are you hoping to gain from this experience?

It would be gratifying to assist at least one associate member of the society in achieving a position in academic research.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 1
            b. Geographic Location - 5
            c. Professional Status / Success - 5
            d. Compatibility of Career Path - 1

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

Dr. Wilber is an Assistant Professor in the Department of Medical Microbiology, Immunology and Cell Biology and Director of the Public Health Laboratory Sciences graduate program at Southern Illinois University School of Medicine. He is a member of American Society of Gene and Cell Therapy and the American Society for Hematology and serves as an academic editor for PLoS On be and a reviewer for Blood, BMC Biology, Stem Cells and PLoS journals. His research efforts are focused on achieving therapeutic levels of the anti-sickling globin in erythroid cells for the treatment sickle cell disease and beta-thalassemia and identifying the role of soble tumor-derived factors on trans-differentiation of natural killer cells.

Click here to view Andy Wilber's Biosketch.

J. Fraser Wright, PhD
Children's Hospital of Philadelphia

What is your current professional status?

Associate Professor of Pathology and Lab Medicine
University of Pennsylvania Perelman School of Medicine
Director, Clinical Vector Core
Center for Cellular and Molecular Therapeutics
Children's Hospital of Philadelphia
3615 Civic Center Blvd, Philadelphia PA 19104

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic institution

What is your scientific area of research?

Translational research in Gene Therapy: Use of recombinant adeno associated viruses (AAV)
and more recently recombinant Lentiviruses (rLenti) to effect therapeutic gene delivery in
humans. Though involved in several projects using rAAV and rLenti gene therapy platforms,
particular focus on gene therapy for hemophilia, congenital blindness, Parkinson’s disease,
and chimeric antigen receptor for cancer immunotherapy.

Why do you want to be a mentor?

I believe that therapeutic gene transfer will have a very significant positive impact on
treatment of important human diseases. I would like to encourage and support young
investigators who are considering or have chosen to develop their careers this important field.

As a mentor, what are you hoping to gain from this experience?

The opportunity to interact, assist and learn from young investigators in the field of gene and
cell therapy, to help advance the field through mentoring of talented young investigators.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

a. Compatibility of Scientific Specialties - 2
b. Geographic Location - 4
c. Professional Status / Success - 3
d. Compatibility of Career Path - 3
e. Communication Style - 5

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

My training and research experience: 1) basic immunology investigating immunoglobulin
structure and function; 2) virology, including immune response, and development of virus
based vaccines and immunotherapeutics; and recombinant adeno-associated virus (rAAV)
and Lentivirus (rLenti) based vectors for gene transfer for human diseases. After post
doctoral studies in molecular immunology and successful establishment of a research
program focused immunology of blood-borne viruses (University of Toronto / CBS), since
1993 my career has focused on translational research using recombinant viral vectors. This
included four years at Sanofi Aventis (recombinant avian poxviruses for human cancer
immunotherapy), five years at Avigen (directing efforts in vector design, development and
cGMP manufacture of AAV vectors for human clinical trials), and since 2004 at The
Children’s Hospital of Philadelphia (CHOP) and University of Pennsylvania Perelman
School of Medicine (established and direct a cGMP clinical vector core facility). Human
clinical trials in which I have played a key role include: AAV expressing human coagulation
factor IX (hFIX) for hemophilia B; AAV expressing aromatic amino acid decarboxylase
(AADC) for Parkinson’s disease; and 3) AAV expressing human retinal pigment epithelium
associated 65kDa protein (RPE65) for Leber Congenital Amaurosis; and immunotherapy
using chimerical antigen receptors. My laboratory is currently the designated NHLBI Gene
Therapy Resource Program AAV Clinical Vector Laboratory, supporting translational
programs by NIH investigators across the country. I have a special interest in human
immunology as it relates to gene therapy.

Click here to view J. Fraser Wright's CV.

Seppo Yla-Herttuala, MD, PhD, FESC
University of Eastern Finland

What is your current professional status?

Professor of Molecular Medicine

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Academic Institution

What is your scientific area of research?

Gene therapy, cardiovascular diseases and vector development

Why do you want to be a mentor?

I am Chairman of the Graduate School in our University's Faculty of Health Sciences and have long experience in mentoring and research training. Therefore, I think that this experience would be very useful for younger scientists.

As a mentor, what are you hoping to gain from this experience?

Helping young scientists to promote their career and also networking with next generation scientists worldwide.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

            a. Compatibility of Scientific Specialties - 2
            b. Geographic Location - 4
            c. Professional Status / Success - 3
            d. Compatibility of Career Path - 1
            e. Communication Style - 3

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

Prof. Yla-Herttuala is internationally recognized leader in cardiovascular gene therapy. His team was the first to use adenovirus-mediated gene transfer in human arteries in 1995. Since then, he has conducted several clinical trials in cardiovascular gene therapy. His research group is also widely recognized for basic biology and applied biology research with vascular endothelial growth factors, especially with the new members of the growth factor family. Professor Yla-Herttuala was President of the European Society of Gene and Cell Therapy 2010-2012 and  is currently a member in the editorial boards of several major US and European cardiovascular and gene therapy journals. His list of publications includes over 350 scientific papers and articles about cardiovascular diseases, VEGFs, atherosclerosis, restenosis and gene therapy.

Seppo Yla-Herttuala's CV coming soon

John A. Zaia, MD
Beckman Research Institute - City of Hope

What is your current professional status?

Chairman, Dept of Virology

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)

Private Research Institute

What is your scientific area of research?

HIV/AIDS gene therapy; viral vaccines; viral immunology

Why do you want to be a mentor?

I find it gratifying and somewhat rejuvenating to work with students/fellows.

As a mentor, what are you hoping to gain from this experience?

I hope to share my advice with a student/fellow.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

a. Compatibility of Scientific Specialties - 2
b. Geographic Location - 5
c. Professional Status / Success - 3
d. Compatibility of Career Path - 2
e. Communication Style - 1

Summarized in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy.

Dr. Zaia specializes in Pediatric Infectious Disease, with a particular interest in infections of immunocompromised patients, especially transplant recipients and HIV/AIDS patients.  His clinical research involves the develop of stem cell approaches to control of HIV/AIDS using gene therapy, and his team was the first to apply such approaches to AIDS lymphoma.  His laboratory studies innate immunity to cytomegalovirus (CMV), and he is the PI on the first evaluation in humans of two novel CMV vaccines developed at COH. 

Click here to view John Zaia's CV.