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Gene & Cell Therapy Breakthroughs
Gene & Cell Therapy Breakthroughs are lay summaries of key Molecular Therapy articles. Each month, beginning in August 2010, the Editor-in-Chief and Managing Editor of Molecular Therapy select one or two manuscripts from Molecular Therapy to be featured in this area of the ASGCT website. The summaries are drafted by the Deputy and Associate Editors of Molecular Therapy. To suggest manuscripts to be featured in Gene & Cell Therapy Breakthroughs, please contact the ASGCT Executive Office.
Promising Caravans of Dreams: Establishing safety of long-term rAAV gene therapy for Canavan’s disease
Guangping Gao, PhD and Seemin S. Ahmed, PhD
Posted: January 29, 2013
Paola Leone1,*,David Shera,Scott W.J. McPhee,
Jeremy S. Francis,Edwin H. Kolodny,Larissa T. Bilaniuk,Dah-Jyuu Wang,Mitra Assadi,Olga Goldfarb,H. Warren Goldman,Andrew Freese,Deborah Young,
Matthew J. During8,9,
R. Jude Samulski3,10 and
Christopher G. Janson7,11
An Old Drug with a New Trick! Glucocorticoids Improve Lentiviral Gene Transfer Into Liver Cells
Michael Ott, MD, Ute Modlich, DVM, PhD, Christopher Baum, MD
Posted: December 04, 2012
A TLR and Non-TLR Mediated Innate Response to Lentiviruses Restricts Hepatocyte Entry and Can be Ameliorated by Pharmacological Blockade
Judith Agudo, Albert Ruzo, Kipyegon Kitur, Ravi Sachidanandam, J Magarian Blander and Brian D Brown
An Antibody to Prevent Antibodies: Toward a Better and Safer Treatment for Pompe Disease
Sushrusha Nayak, PhD, and Roland W. Herzog, PhD
Posted: October 22, 2012
Administration of Anti-CD3 Antibodies Modulates the Immune Response to an Infusion of á-glucosidase in Mice
Toya Ohashi, Sayoko Iizuka, Yohta Shimada, Takashi Higuchi, Yoshikatsu Eto, Hiroyuki Ida1
and Hiroshi Kobayashi
A New Way to Patch Up Broken Hearts: Repeated Sphere Formation
Elena Senis & Dirk Grimm, PhD
Posted: August 22, 2012
Secondary Sphere Formation Enhances the Functionality of Cardiac Progenitor Cells
Hyun-Jai Cho,Ho-Jae Lee Seock-Won Youn, Seok-Jin Koh, Joo-Yun Won, Yeon-Ju Chung,
Hyun-Ju Cho, Chang-Hwan Yoon, Sae-Won Lee, Eun Ju Lee, Yoo-Wook Kwon, Hae-Young Lee,
Sang Hun Lee4, Won-Kyung Ho4, Young-Bae Park1,2 and Hyo-Soo Kim1,2,3,5
An Alternative Treatment to Lung Transplantation.
Jacques P. Tremblay, PhD
November 29, 2012
Fighting off viral infections after hematopoietic stem cell transplantation: A single treatment that hits many targets.
Dr. Roland R. Herzog, PhD
August 8, 2012
New Cells for Old Limbs
Nicole M Kane PhD.
June 27, 2012
Promising Safety Profile: Gene therapy Using AAV-Based Delivery Shuttles
Jeffrey S. Chamberlain, PhD
June 19, 2012
SHAPE Really Matters When it Comes to Short Hairpin RNAs
John J. Rossi, PhD
April 18, 2012
Treating Muscular Dystrophy with Membrane Resealing
Dongsheng Duan, PhD
April 6, 2012
Dual Function in vivo Delivery of an Inhibitory miRNA and a Corrective Protein Encoding Gene
John J. Rossi, PhD
March 1, 2012
A novel processing pathway for microRNA molecules with therapeutic potential
Lynda Coughlan, PhD
February 2, 2012
Duchenne Muscular Dystrophy: on a rational road to the cure?
Roland W. Herzog, PhD and Irene Zolotukhin, MS
February 2, 2012
Liver Cells Derived from Stem Cells Will be Used to Test New Drugs
Jacques P. Tremblay, PhD
December 28, 2011
Targeting T Cell-Mediated Immune Response in Malaria Vaccination
Roland W. Herzog, PhD and Xiaomei Wang, PhD
November 18, 2011
Systemic Delivery Following Pulmonary Administration of Large-Molecule Nucleic Acid Drugs
Mark A. Behlke, MD, PhD
November 18, 2011
Triggering Anti-Tumor Killing with Aptamers
John J. Rossi, PhD
October 12, 2011
A Tractable Model of Human Cardiac Development
Nicole M. Kane, PhD
September 26, 2011
Buldging at the Seeds Abrogates Off-Targeting of siRNAs
John J. Rossi, PhD
September 23, 2011
Smart Bombs for Targeted Delivery of Therapeutic Small RNAs
John J. Rossi, PhD
July 12, 2011
Gene Therapy for Prostate Cancer - A New Therapeutic Hope?
Alan L. Parker, PhD
July 7, 2011
Magic Bullet Targeting Subcellular Organelle: Mitochondria
Yuhua Wang, PhD and Leaf Huang, PhD
July 7, 2011
Curing Sick Stem Cells via Gene Therapy
Stuart A. Nicklin, PhD
June 7, 2011
Switching on the Lights in Blind Eyes
Jean Bennett, MD, PhD and Therese Cronin, PhD
June 2, 2011
Repairing the Heart with Bacteria?
Andrew H. Baker, PhD
May 5, 2011
Gene-modified stem cells: the next generation therapy for complex bone fractures
Dan Gazit, DMD, PhD
May 5, 2011
Gene Vector Integration Profiling in Eye and Brain
Christopher Baum, MD
April 27, 2011
Treating Bowel Cancer with Gene Therapy: Too Much of a Good Thing
Malcolm K. Brenner, MD, PhD
April 4, 2011
When going viral, the right model makes all the difference
David Markusic, PhD and Roland Herzog, PhD
February 11, 2011
A need to teach tumor-killing viruses not to suppress the immune system
George Q. Perrin, PhD, and Roland W. Herzog, PhD
January 7, 2011
Safety Improvements for Stem Cell "Taxi Cab"
Malcolm K. Brenner, MD, PhD
December 1, 2010
Tricking the immune system into tolerating a therapeutic protein
Roland W. Herzog, PhD
December 1, 2010
Harnessing Stem Cells for Regenerative Medicine
Lonnie D. Shea, PhD
November 8, 2010
A Pox on Your Brain Tumor
Timothy Cripe, MD, PhD
November 1, 2010
Restoring Muscle Strength in DMD Mice
Jacques P. Tremblay, PhD
October 6, 2010
Testing a Painless Treatment for Skin Diseases
John J. Rossi, PhD
September 9, 2010
One Plus One Equals Three in Gene Therapy for Cancer Study
John C. Bell, PhD
August 2, 2010