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Article ; Online: Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials.

Chamberlain, Jeffrey S / Robb, Melissa / Braun, Serge / Brown, Kristy J / Danos, Olivier / Ganot, Annie / Gonzalez-Alegre, Pedro / Hunter, Nina / McDonald, Craig / Morris, Carl / Tobolowsky, Mark / Wagner, Kathryn R / Ziolkowski, Olivia / Duan, Dongsheng

Human gene therapy

2023  Volume 34, Issue 9-10, Page(s) 404–415

Abstract: Duchenne muscular dystrophy (DMD) is a serious, rare genetic disease, affecting primarily boys. It is caused by mutations in ... ...

Abstract Duchenne muscular dystrophy (DMD) is a serious, rare genetic disease, affecting primarily boys. It is caused by mutations in the
MeSH term(s) Male ; Humans ; Muscular Dystrophy, Duchenne/genetics ; Muscle, Skeletal/metabolism ; Genetic Therapy/methods ; Gene Transfer Techniques ; Biomarkers/metabolism
Chemical Substances Biomarkers
Language English
Publishing date 2023-03-14
Publishing country United States
Document type Journal Article ; Research Support, Non-U.S. Gov't ; Research Support, N.I.H., Extramural
ZDB-ID 1028152-6
ISSN 1557-7422 ; 1043-0342
ISSN (online) 1557-7422
ISSN 1043-0342
DOI 10.1089/hum.2022.190
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Zs.A 2988: Show issues Location:
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