Sage Journals: Discover world-class research

Retricted access

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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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

Open access

Research article

First published 2021-04

Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy

Rachael A. Potter, Danielle A. Griffin, Kristin N. Heller [...]

Human Gene Therapy

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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

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Research article

First published 2023-05

Lifelong Outcomes of Systemic Adeno-Associated Virus Micro-Dystrophin Gene Therapy in a Murine Duchenne Muscular Dystrophy Model

Nalinda B. Wasala, Yongping Yue, Bryan Hu [...]

Human Gene Therapy

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Research article

First published 2005-1

Development of a Lupus Anti-Dna Antibody as An Intracellular Delivery Vehicle for Protein Transduction of Micro-Dystrophin

R.H. Weisbart, G. Chan, R. Wakelin [...]

Journal of Investigative Medicine

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Other

First published 2010-9

Preclinical Studies for Gene Therapy of Duchenne Muscular Dystrophy

Guy L. Odom, Glen B. Banks, Brian R. Schultz [...]

Journal of Child Neurology

Open access

Brief report

First published 2021-07-30

Evaluation of the Lipid-binding Properties of Recombinant Dystrophin Spectrin-like Repeat Domains R1-3

Grace Cooper-Olson

Journal of Neuromuscular Diseases

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Research article

First published 2018-07

Cardiac-Specific Expression of ΔH2-R15 Mini-Dystrophin Normalized All Electrocardiogram Abnormalities and the End-Diastolic Volume in a 23-Month-Old Mouse Model of Duchenne Dilated Cardiomyopathy

Nalinda B. Wasala, Jin-Hong Shin, Yi Lai [...]

Human Gene Therapy

Open access

Article commentary

First published 2023-05-02

The Dilemma of Choice for Duchenne Patients Eligible for Exon 51 Skipping The European Experience

Annemieke Aartsma-Rus, Liesbeth De Waele, Saskia Houwen-Opstal [...]

Journal of Neuromuscular Diseases

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