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

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...

Full description

Bibliographic Details
Main Authors: Tabatha R. Simmons, Tatyana A. Vetter, Nianyuan Huang, Adeline Vulin-Chaffiol, Nicolas Wein, Kevin M. Flanigan
Format: Article
Language:English
Published: Elsevier 2021-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Duchenne muscular dystrophy
Becker muscular dystrophy
dystrophin
exon skipping
U7snRNA
gene therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121000541

Internet

http://www.sciencedirect.com/science/article/pii/S2329050121000541

Similar Items