A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model...
A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy
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Publisher
United States: Elsevier Limited
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Language
English
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United States: Elsevier Limited
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Contents
Micro-dystrophins are highly promising candidates for treating Duchenne muscular dystrophy, a lethal muscle disease caused by dystrophin deficiency. Here, we report robust disease rescue in the severe DBA/2J-mdx model with a neuronal nitric oxide synthase (nNOS)-binding micro-dystrophin vector. 2 × 10
vector genome particles/mouse of the vector...
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Full title
A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy
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TN_cdi_doaj_primary_oai_doaj_org_article_a968d82e3a484f8cb6cd48ebc8ad7bec
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_a968d82e3a484f8cb6cd48ebc8ad7bec
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ISSN
2329-0501
E-ISSN
2329-0501
DOI
10.1016/j.omtm.2017.06.006
