Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping

Duchenne muscular dystrophy (DMD) is an X-linked progressive disease characterized by loss of dystrophin protein that typically results from truncating mutations in the DMD gene. Current exon-skipping therapies have sought to treat deletion mutations that abolish an open reading frame (ORF) by skipp...

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Hlavní autoři:	Tabatha R. Simmons, Tatyana A. Vetter, Nianyuan Huang, Adeline Vulin-Chaffiol, Nicolas Wein, Kevin M. Flanigan
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Elsevier 2021-06-01
Edice:	Molecular Therapy: Methods & Clinical Development
Témata:	Duchenne muscular dystrophy Becker muscular dystrophy dystrophin exon skipping U7snRNA gene therapy
On-line přístup:	http://www.sciencedirect.com/science/article/pii/S2329050121000541
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