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Molecular Therapy. Methods & Clinical Development
|
June 2, 2015
Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxidative damage to transgene mRNA
Jean-Baptiste Dupont, Benoit Tournaire, Christophe Georger, et al.
Molecular Therapy. Methods & Clinical Development
|
May 2, 2020
Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
Karim Bey, Johan Deniaud, Laurence Dubreil, et al.
Journal of Neuromuscular Diseases
|
November 19, 2016
Non-Ambulant Duchenne Patients Theoretically Treatable by Exon 53 Skipping have Severe Phenotype
Laurent Servais, Marie Montus, Caroline Le Guiner, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 24, 2014
Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates
Marinee K Chuah, Inge Petrus, Pieter De Bleser, et al.
Molecular Therapy. Methods & Clinical Development
|
February 8, 2024
Mannose-coupled AAV2: A second-generation AAV vector for increased retinal gene therapy efficiency
Mathieu Mével, Virginie Pichard, Mohammed Bouzelha, et al.
Gene Therapy
|
February 2, 2022
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMD<sup>mdx</sup> rat model
Audrey Bourdon, Virginie François, Liwen Zhang, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
December 9, 2010
Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes
N Matthew Ellinwood, Jérôme Ausseil, Nathalie Desmaris, et al.
Science Translational Medicine
|
January 24, 2014
Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy
Martin K Childers, Romain Joubert, Karine Poulard, 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.
Human Gene Therapy
|
October 3, 2014
Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material
Eduard Ayuso, Véronique Blouin, Martin Lock, et al.
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of 10
Search research articles
Search
Showing results (81-90 of 92) with videos related to
Sort By:
Page
of 10
Molecular Therapy. Methods & Clinical Development
|
June 2, 2015
Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxidative damage to transgene mRNA
Jean-Baptiste Dupont, Benoit Tournaire, Christophe Georger, et al.
Molecular Therapy. Methods & Clinical Development
|
May 2, 2020
Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
Karim Bey, Johan Deniaud, Laurence Dubreil, et al.
Journal of Neuromuscular Diseases
|
November 19, 2016
Non-Ambulant Duchenne Patients Theoretically Treatable by Exon 53 Skipping have Severe Phenotype
Laurent Servais, Marie Montus, Caroline Le Guiner, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 24, 2014
Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates
Marinee K Chuah, Inge Petrus, Pieter De Bleser, et al.
Molecular Therapy. Methods & Clinical Development
|
February 8, 2024
Mannose-coupled AAV2: A second-generation AAV vector for increased retinal gene therapy efficiency
Mathieu Mével, Virginie Pichard, Mohammed Bouzelha, et al.
Gene Therapy
|
February 2, 2022
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMD<sup>mdx</sup> rat model
Audrey Bourdon, Virginie François, Liwen Zhang, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
December 9, 2010
Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes
N Matthew Ellinwood, Jérôme Ausseil, Nathalie Desmaris, et al.
Science Translational Medicine
|
January 24, 2014
Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy
Martin K Childers, Romain Joubert, Karine Poulard, 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.
Human Gene Therapy
|
October 3, 2014
Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material
Eduard Ayuso, Véronique Blouin, Martin Lock, et al.
Page
of 10