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Research article

First published 1998-03

High-Level Dystrophin Expression after Adenovirus-Mediated Dystrophin Minigene Transfer to Skeletal Muscle of Dystrophic Dogs: Prolongation of Expression with Immunosuppression

John McC. Howell, Hanns Lochmüller, Amanda O'Hara [...]

Human Gene Therapy

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Research article

First published 1995-11

Recombinant Truncated Dystrophin Minigenes: Construction,Expression,and Adenoviral Delivery

Paula R. Clemens, Todd L. Krause, Suzanne Chan [...]

Human Gene Therapy

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Research article

First published 2004-08

Intraarterial Delivery of Naked Plasmid DNA Expressing Full-Length Mouse Dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy

Goufeng Zhang, James J. Ludtke, Christine Thioudellet [...]

Human Gene Therapy

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Research article

First published 2004-08

Chimeric RNA/Ethylene-Bridged Nucleic Acids Promote Dystrophin Expression in Myocytes of Duchenne Muscular Dystrophy by Inducing Skipping of the Nonsense Mutation-Encoding Exon

Agus Surono, Tran Van Khanh, Yasuhiro Takeshima [...]

Human Gene Therapy

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Research article

First published 2021-02

Transplantation of Dystrophin Expressing Chimeric Human Cells of Myoblast/Mesenchymal Stem Cell Origin Improves Function in Duchenne Muscular Dystrophy Model

Maria Siemionow, Erzsebet Szilagyi, Joanna Cwykiel [...]

Stem Cells and Development

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Research article

First published 2012-02

A Simplified Immune Suppression Scheme Leads to Persistent Micro-dystrophin Expression in Duchenne Muscular Dystrophy Dogs

Jin-Hong Shin, Yongping Yue, Arun Srivastava [...]

Human Gene Therapy

Open access

Research article

First published 2010-02

Functional Efficacy of Dystrophin Expression from Plasmids Delivered to mdx Mice by Hydrodynamic Limb Vein Injection

Guofeng Zhang, Christine I. Wooddell, Julia O. Hegge [...]

Human Gene Therapy

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Research article

First published 1996-01

Dystrophin Expression in Muscles of mdx Mice After Adenovirus-Mediated In Vivo Gene Transfer

Gyula Acsadi, Hanns Lochmüller, Agnes Jani [...]

Human Gene Therapy

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Research article

First published 2008-08

Functionalized PEG–PEI Copolymers Complexed to Exon-Skipping Oligonucleotides Improve Dystrophin Expression in mdx Mice

Shashank R. Sirsi, Rebecca C. Schray, Xiangying Guan [...]

Human Gene Therapy

Open access

Research article

First published 2025-04

BMN 351-Induced Exon Skipping and Dystrophin Expression in Skeletal and Cardiac Muscle Lead to Preservation of Motor Function in a Mouse Model of Exon 51 Skip-Amenable Duchenne Muscular Dystrophy

Todd Oppeneer, Yulan Qi, Joshua Henshaw [...]

Nucleic Acid Therapeutics

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