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Journal of Neuromuscular Diseases
|
March 29, 2021
Evaluation of the Lipid-binding Properties of Recombinant Dystrophin Spectrin-like Repeat Domains R1-3
Grace Cooper-Olson, Louise R Rodino-Klapac, Rachael A Potter
Biology Open
|
September 6, 2023
Expression and function of four AAV-based constructs for dystrophin restoration in the mdx mouse model of Duchenne muscular dystrophy
Rachael A Potter, Danielle A Griffin, Kristin N Heller, et al.
Human Gene Therapy
|
December 22, 2020
Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy
Danielle A Griffin, Eric R Pozsgai, Kristin N Heller, et al.
Expert Opinion on Biological Therapy
|
February 8, 2020
Clinical development on the frontier: gene therapy for duchenne muscular dystrophy
Damon R Asher, Khampaseuth Thapa, Sachi D Dharia, et al.
Human Gene Therapy
|
January 5, 2021
Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the <i>mdx</i> Mouse Model of Duchenne Muscular Dystrophy
Rachael A Potter, Danielle A Griffin, Kristin N Heller, et al.
Molecular Therapy. Methods & Clinical Development
|
February 8, 2024
Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74
Rachael A Potter, Ellyn L Peterson, Danielle Griffin, et al.
BMC Cell Biology
|
September 24, 2013
Suppression of protein kinase C theta contributes to enhanced myogenesis in vitro via IRS1 and ERK1/2 phosphorylation
Joseph S Marino, Terry D Hinds, Rachael A Potter, et al.
Human Gene Therapy
|
November 28, 2024
Long-Term Survival and Myocardial Function Following Systemic Delivery of Delandistrogene Moxeparvovec in DMD<sup>MDX</sup> Rats
Stephen Baine, Chris Wier, Luke Lemmerman, et al.
Molecular Therapy. Methods & Clinical Development
|
February 23, 2023
Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model
Young-Eun Seo, Stephen H Baine, Amber N Kempton, et al.
Scientific Reports
|
January 3, 2025
Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy
Rachael A Potter, Ida H Moeller, Sohrab Khan, et al.
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Search research articles
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Showing results (1-10 of 16) with videos related to
Sort By:
Page
of 2
Journal of Neuromuscular Diseases
|
March 29, 2021
Evaluation of the Lipid-binding Properties of Recombinant Dystrophin Spectrin-like Repeat Domains R1-3
Grace Cooper-Olson, Louise R Rodino-Klapac, Rachael A Potter
Biology Open
|
September 6, 2023
Expression and function of four AAV-based constructs for dystrophin restoration in the mdx mouse model of Duchenne muscular dystrophy
Rachael A Potter, Danielle A Griffin, Kristin N Heller, et al.
Human Gene Therapy
|
December 22, 2020
Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy
Danielle A Griffin, Eric R Pozsgai, Kristin N Heller, et al.
Expert Opinion on Biological Therapy
|
February 8, 2020
Clinical development on the frontier: gene therapy for duchenne muscular dystrophy
Damon R Asher, Khampaseuth Thapa, Sachi D Dharia, et al.
Human Gene Therapy
|
January 5, 2021
Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the <i>mdx</i> Mouse Model of Duchenne Muscular Dystrophy
Rachael A Potter, Danielle A Griffin, Kristin N Heller, et al.
Molecular Therapy. Methods & Clinical Development
|
February 8, 2024
Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74
Rachael A Potter, Ellyn L Peterson, Danielle Griffin, et al.
BMC Cell Biology
|
September 24, 2013
Suppression of protein kinase C theta contributes to enhanced myogenesis in vitro via IRS1 and ERK1/2 phosphorylation
Joseph S Marino, Terry D Hinds, Rachael A Potter, et al.
Human Gene Therapy
|
November 28, 2024
Long-Term Survival and Myocardial Function Following Systemic Delivery of Delandistrogene Moxeparvovec in DMD<sup>MDX</sup> Rats
Stephen Baine, Chris Wier, Luke Lemmerman, et al.
Molecular Therapy. Methods & Clinical Development
|
February 23, 2023
Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model
Young-Eun Seo, Stephen H Baine, Amber N Kempton, et al.
Scientific Reports
|
January 3, 2025
Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy
Rachael A Potter, Ida H Moeller, Sohrab Khan, et al.
Page
of 2