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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
February 27, 2017
Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs
David L Mack, Karine Poulard, Melissa A Goddard, et al.
Human Gene Therapy
|
February 6, 2015
Perspectives on best practices for gene therapy programs
Thomas R Cheever, Dale Berkley, Serge Braun, et al.
Nature Medicine
|
December 8, 2009
Comprehensive genomic access to vector integration in clinical gene therapy
Richard Gabriel, Ralph Eckenberg, Anna Paruzynski, et al.
Nature Communications
|
April 1, 2025
Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease
Steicy Sobrino, Laure Joseph, Elisa Magrin, et al.
Nature Communications
|
July 26, 2017
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Caroline Le Guiner, Laurent Servais, Marie Montus, et al.
JAMA
|
April 22, 2015
Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome
Salima Hacein-Bey Abina, H Bobby Gaspar, Johanna Blondeau, et al.
The Lancet. Neurology
|
November 17, 2023
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
Perry B Shieh, Nancy L Kuntz, James J Dowling, et al.
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Search research articles
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Showing results (81-90 of 87) with videos related to
Sort By:
Page
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You have reached the last page of results.
This site can display upto 87 results.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
February 27, 2017
Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs
David L Mack, Karine Poulard, Melissa A Goddard, et al.
Human Gene Therapy
|
February 6, 2015
Perspectives on best practices for gene therapy programs
Thomas R Cheever, Dale Berkley, Serge Braun, et al.
Nature Medicine
|
December 8, 2009
Comprehensive genomic access to vector integration in clinical gene therapy
Richard Gabriel, Ralph Eckenberg, Anna Paruzynski, et al.
Nature Communications
|
April 1, 2025
Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease
Steicy Sobrino, Laure Joseph, Elisa Magrin, et al.
Nature Communications
|
July 26, 2017
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Caroline Le Guiner, Laurent Servais, Marie Montus, et al.
JAMA
|
April 22, 2015
Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome
Salima Hacein-Bey Abina, H Bobby Gaspar, Johanna Blondeau, et al.
The Lancet. Neurology
|
November 17, 2023
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
Perry B Shieh, Nancy L Kuntz, James J Dowling, et al.
Page
of 9