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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
September 27, 2012
Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice
Kamal K E Gadalla, Mark E S Bailey, Rosemary C Spike, et al.
Science Translational Medicine
|
December 21, 2012
Long-term follow-up after gene therapy for canavan disease
Paola Leone, David Shera, Scott W J McPhee, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 10, 2011
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector
Dawn E Bowles, Scott W J McPhee, Chengwen Li, et al.
Human Gene Therapy
|
November 25, 2014
Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial
Paul E Monahan, Junjiang Sun, Tong Gui, et al.
Journal of the American College of Cardiology
|
September 30, 2017
Protein Phosphatase Inhibitor-1 Gene Therapy in a Swine Model of Nonischemic Heart Failure
Shin Watanabe, Kiyotake Ishikawa, Kenneth Fish, et al.
The New England Journal of Medicine
|
October 8, 2010
Dystrophin immunity in Duchenne's muscular dystrophy
Jerry R Mendell, Katherine Campbell, Louise Rodino-Klapac, et al.
Blood
|
October 17, 2020
BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression
Barbara A Konkle, Christopher E Walsh, Miguel A Escobar, et al.
Journal of Virology
|
June 14, 2013
Capsid antibodies to different adeno-associated virus serotypes bind common regions
Brittney L Gurda, Michael A DiMattia, Edward B Miller, et al.
Nature Medicine
|
October 21, 2025
Cardiotropic AAV gene therapy for heart failure: a phase 1 trial
Timothy D Henry, Eugene S Chung, Monica Alvisi, et al.
Human Gene Therapy
|
March 23, 2013
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012
Marina O'Reilly, Donald B Kohn, Jeffrey Bartlett, et al.
Page
of 23
Search research articles
Search
Showing results (211-220 of 221) with videos related to
Sort By:
Page
of 23
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
September 27, 2012
Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice
Kamal K E Gadalla, Mark E S Bailey, Rosemary C Spike, et al.
Science Translational Medicine
|
December 21, 2012
Long-term follow-up after gene therapy for canavan disease
Paola Leone, David Shera, Scott W J McPhee, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 10, 2011
Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector
Dawn E Bowles, Scott W J McPhee, Chengwen Li, et al.
Human Gene Therapy
|
November 25, 2014
Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial
Paul E Monahan, Junjiang Sun, Tong Gui, et al.
Journal of the American College of Cardiology
|
September 30, 2017
Protein Phosphatase Inhibitor-1 Gene Therapy in a Swine Model of Nonischemic Heart Failure
Shin Watanabe, Kiyotake Ishikawa, Kenneth Fish, et al.
The New England Journal of Medicine
|
October 8, 2010
Dystrophin immunity in Duchenne's muscular dystrophy
Jerry R Mendell, Katherine Campbell, Louise Rodino-Klapac, et al.
Blood
|
October 17, 2020
BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression
Barbara A Konkle, Christopher E Walsh, Miguel A Escobar, et al.
Journal of Virology
|
June 14, 2013
Capsid antibodies to different adeno-associated virus serotypes bind common regions
Brittney L Gurda, Michael A DiMattia, Edward B Miller, et al.
Nature Medicine
|
October 21, 2025
Cardiotropic AAV gene therapy for heart failure: a phase 1 trial
Timothy D Henry, Eugene S Chung, Monica Alvisi, et al.
Human Gene Therapy
|
March 23, 2013
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012
Marina O'Reilly, Donald B Kohn, Jeffrey Bartlett, et al.
Page
of 23