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Research article

First published 2023-05

Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials

Jeffrey S. Chamberlain, Melissa Robb, Serge Braun [...]

Human Gene Therapy

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Brief report

First published 2008-08

Adeno-Associated Virus Serotype-9 Microdystrophin Gene Therapy Ameliorates Electrocardiographic Abnormalities in mdx Mice

Brian Bostick, Yongping Yue, Yi Lai [...]

Human Gene Therapy

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Brief report

First published 2009-06

Efficient and Fast Functional Screening of Microdystrophin Constructs In Vivo and In Vitro for Therapy of Duchenne Muscular Dystrophy

Louise H. Jørgensen, Nancy Larochelle, Kristian Orlopp [...]

Human Gene Therapy

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Research article

First published 2007-06

Herpes Simplex Virus VP22 Enhances Adenovirus-Mediated Microdystrophin Gene Transfer to Skeletal Muscles in Dystrophin-Deficient ( mdx ) Mice

Fu Xiong, Shaobo Xiao, Funing Peng [...]

Human Gene Therapy

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Research article

First published 2021-10

Dose-Dependent Microdystrophin Expression Enhancement in Cardiac Muscle by a Cardiac-Specific Regulatory Element

Alberto Malerba, Chiara Sidoli, Ngoc Lu-Nguyen [...]

Human Gene Therapy

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Research article

First published 2011-11

Delivery of AAV2/9-Microdystrophin Genes Incorporating Helix 1 of the Coiled-Coil Motif in the C-Terminal Domain of Dystrophin Improves Muscle Pathology and Restores the Level of α1-Syntrophin and α-Dystrobrevin in Skeletal Muscles of mdx Mice

Taeyoung Koo, Alberto Malerba, Takis Athanasopoulos [...]

Human Gene Therapy

Free access

Research article

First published 2021-11-30

The Effect of Immunomodulatory Treatments on Anti-Dystrophin Immune Response After AAV Gene Therapy in Dystrophin Deficient mdx Mice

Ning Li, Joanna E. Parkes, Rita Spathis [...]

Journal of Neuromuscular Diseases

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Research article

First published 2025-09

Periostin Exon 17 Skipping Enhances the Efficacy of Local Adeno-Associated Viral-Microdystrophin Administration in a Fibrotic Model of Duchenne Muscular Dystrophy

Jessica Trundle, Alexis Boulinguiez, Ngoc Lu-Nguyen [...]

Human Gene Therapy

Open access

Review article

First published 2021-11-30

Gene Therapy for Duchenne Muscular Dystrophy

Nertiyan Elangkovan, George Dickson

Journal of Neuromuscular Diseases

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Research article

First published 2012-06

Long-Term Preservation of Cardiac Structure and Function After Adeno-Associated Virus Serotype 9-Mediated Microdystrophin Gene Transfer in mdx Mice

Stefanie Schinkel, Ralf Bauer, Raffi Bekeredjian [...]

Human Gene Therapy

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