Micro-dystrophin Genes Bring Hope of an Effective Therapy for Duchenne Muscular Dystrophy
Author:
Funder
Medical Research Council
Publisher
Elsevier BV
Subject
Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine
Reference16 articles.
1. Progress toward Gene Therapy for Duchenne Muscular Dystrophy;Chamberlain;Mol Ther.,2017
2. Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy;Duan;Mol Ther.,2018
3. Development of Novel Micro-dystrophins with Enhanced Functionality;Ramos;Mol Ther.,2019
4. The Pathogenesis and Therapy of Muscular Dystrophies;Guiraud;Annu. Rev. Genomics Hum. Genet.,2015
5. Very mild muscular dystrophy associated with the deletion of 46% of dystrophin;England;Nature,1990
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