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Special Media Program - "Horizons in Gene Therapy"
On Thursday, May 31, 2007The American Society of Gene Therapy (ASGT) invited members of the press to a media only event designed to highlight some of the latest and most promising developments in gene therapy products and related technologies.
Several children born with "bubble-boy disease" caused by ADA deficiency have been cured by gene therapy without major side effects and researchers have ameliorated a growing list of disease conditions in animals including diabetes, arthritis, heart disease and cancer. Some of these gene therapy treatments are now in advanced stages of clinical trials.
Leading scientists in the fields of cancer, arthritis, infectious diseases, and childhood blindness discussed their research and progress toward bringing these promising gene therapy technologies and products to the clinic. Panelists included:
Click on the presentation title following the name of each presenter, to view their slides from the program "Horizons in Gene Therapy." If you would like to view the video presentations rather than the slides only version, go to the link at the bottom of this page.
Introduction
Dr. Friedmann is Professor of Pediatrics and Director of the Center for Molecular Genetics at University of California San Diego School of Medicine, and current president of the American Society of Gene Therapy. Dr. Friedmann has been a leader in the field of gene-based therapies for over more than three decades. He is widely credited for helping to bring the potential of this field to theforefront when he wrote a seminal paper called "Gene Therapy for Human Genetic Disease?" that appeared in the journal Science in 1972. His expertise and research interests are in the development of model systems for human gene therapy, with techniques including gene isolation and characterization, constructions of viral vectors and in-vivo replacement of genetically altered cells. Dr. Friedmann received his M.D. from the University of Pennsylvania, where he was recently honored with a 2006 Distinguished Graduate Award.
 Joseph C. Glorioso III , Ph.D. Gene Therapy: Overview
Dr. Glorioso is chairman of the department of molecular genetics and biochemistry at the University of Pittsburgh School of Medicine. He is world-renowned for his work on the molecular and genetic aspects of the herpes simplex virus and how to better engineer this organism as a vector for transporting therapeutic genes. Through Dr. Glorioso's initiative, the department of molecular genetics and biochemistry has gained an international reputation for its expertise in engineering and applying different viral vector systems for gene therapy for conditions such as muscular dystrophy, arthritis, inherited metabolic disorders, chronic pain, cancer, juvenile diabetes and cardiovascular disease. He is former president of the American Society of Gene Therapy and a current advisory board member. He is currently the USA editor for the journal Gene Therapy. Dr. Glorioso received his Ph.D. in microbiology from Louisiana State University and did his post-doctoral training at Louisiana State University Medical Center.
 Kristen M. Hege, M.D. GVAX Immunotherpay For Prostate Cancer
Dr. Hege is vice president of clinical research at Cell Genesys. She first joined Cell Genesys in January of 1994 as a post-doctoral research fellow, working on preclinical studies of hematopoietic stem cell gene therapy. For the past10years she has worked in the clinical research department, most recently asvice presidentof clinical research. Dr. Hege participated in the first clinical trial ever performed at the company and since then has overseen many of the clinical programs, including GVAX® cancerimmunotherapies,T cell gene therapy for cancer and HIV infection, andselectively replicatingviral therapies for cancer. In addition to her work at Cell Genesys, Dr. Hege has held a clinical faculty appointment at UCSF since 1997 in the adult leukemia and bone marrow transplant program. Dr. Hege received an M.D. from the University of California, San Francisco. She is a member of the Phi Beta Kappa and Alpha Omega Alpha academic honor societies. She received her subspecialty training at Harvard Medical School and UCSF and is board certified in Hematology and Oncology.
 Philip J. Mease, M.D. Gene Therapy For Arthritis
Dr. Mease is a clinical professor at the University of Washington School of Medicine in Seattle and chief of rheumatology clinical research at the Swedish Hospital Medical Center. He conducts clinical practice with Seattle Rheumatology Associates. His primary research interests include pain and rheumatologic and inflammatory diseases, and conducts clinical trials of emerging therapies for a number of rheumatic disease conditions, including rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, lupus, osteoarthritis, fibromyalgia and osteoporosis. He has authored numerous articles and book chapters and is on review boards for Arthritis & Rheumatism, The Journal of Rheumatology, and The Annals of the Rheumatic Diseases. Awards he has received include the Medical Communicator Award of the American College of Rheumatology. Dr. Mease earned his MD from Stanford University Medical School. He completed his residency in internal medicine at the University of Washington School of Medicine, where he was subsequently chief resident and fellow in rheumatology.
 John Rossi, Ph.D. Combinatorial RNA Based Gene Therapy for the Treatment of HIV Infection
Dr. Rossi is Associate Director for Laboratory Research, Comprehensive Cancer Center and Co-leader, Cancer Biology Program, at the City of Hope, Beckman Research Institute. He also is dean of the Graduate School of Biological Sciences and
Lidow Family Research Chair and Professor, Division of Molecular Biology. A specific goal of his laboratory research is inhibition of HIV infection in patients. To this end, his laboratory is developing gene therapy vectors that can be used to transduce human hematopoietic stem cells with ribozymes and other inhibitory RNA based gene constructs. His laboratory, in collaboration with the laboratory of John A. Zaia, MD, at City of Hope, and Don Kohn, MD, at Childrens’ Hospital of Los Angeles, is engaged in the first human trial using ribozymes in human hematopoietic stem cell-based gene therapy. Future vectors will incorporate siRNA genes and nucleolar localized anti-HIV ribozymes and decoy RNAs. Dr. Rossi received his Ph.D. in microbial genetics from the University of Connecticut and did his postdoctoral training at Brown University.
 Jean Bennett, M.D., Ph.D. Gene Therapy for Congenital Blindness
Dr. Bennett is Professor, Ophthalmology, Cell and Developmental Biology, University of Pennsylvania School of Medicine. She studies the molecular genetics of inherited retinal degenerations with the idea of using this knowledge to develop rational approaches for treatment of these diseases. Target diseases include retinitis pigmentosa and age-related macular degeneration. Studies in her laboratory range for identifying the molecular bases of retinal degenerations, generating animal models for these diseases and rescuing vision in animal models through gene based treatments. Dr. Bennett was one of the first investigators to use viral vectors to deliver transgenes to specific cells in the retina and also led the first team to demonstrate proof-of-principle of ocular gene therapy. She has developed a number of strategies for gene therapy-mediated treatments for retinal disease. She received her Ph.D. from the University of California, Berkeley, and her M.D. from Harvard Medical School.
 View "Horizons in Gene Therapy" Lectures from the 10th Annual Meeting
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