Sage Journals: Discover world-class research

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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Other

First published 2007-07

Novel Mutation in Splicing Donor of Dystrophin Gene First Exon in a Patient With Dilated Cardiomyopathy but No Clinical Signs of Skeletal Myopathy

Shigemi Kimura, Makoto Ikezawa, Shiro Ozasa [...]

Journal of Child Neurology

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

Open access

Research article

First published 2016-2

Distribution of dystrophin gene deletions in a Chinese population

Yuanyuan Li, Zhuo Liu, Shengrong OuYang [...]

Journal of International Medical Research

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Review article

First published 2018-07

Micro-Dystrophin Gene Therapy Goes Systemic in Duchenne Muscular Dystrophy Patients

Dongsheng Duan

Human Gene Therapy

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Other

First published 2016-03

Dystrophin Gene Replacement and Gene Repair Therapy for Duchenne Muscular Dystrophy in 2016: An Interview

Dongsheng Duan

Human Gene Therapy Clinical Development

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Research article

First published 2018-03

Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine Model

Kasun Kodippili, Chady H. Hakim, Xiufang Pan [...]

Human Gene Therapy

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Research article

First published 2004-11

Phase I Study of Dystrophin Plasmid-Based Gene Therapy in Duchenne/Becker Muscular Dystrophy

Norma B. Romero, Serge Braun, Olivier Benveniste [...]

Human Gene Therapy

Open access

Research article

First published 2024-04-30

Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy

Craig M. Zaidman, Natalie L. Goedeker, Amal A. Aqul [...]

Journal of Neuromuscular Diseases

Retricted access

Research article

First published 2023-05

The Implication of Hinge 1 and Hinge 4 in Micro-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy

Lakmini P. Wasala, Thais B. Watkins, Nalinda B. Wasala [...]

Human Gene Therapy

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