Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes
Author:
Funder
Friends of FSH Research
National Institute of Arthritis and Musculoskeletal and Skin Diseases
Publisher
Elsevier BV
Subject
Drug Discovery,Molecular Medicine
Reference61 articles.
1. Population-based incidence and prevalence of facioscapulohumeral dystrophy;Deenen;Neurology,2014
2. Atypical onset in a series of 122 cases with FacioScapuloHumeral Muscular Dystrophy;Pastorello;Clin. Neurol. Neurosurg.,2012
3. Severe phenotype in infantile facioscapulohumeral muscular dystrophy;Klinge;Neuromuscul. Disord.,2006
4. Facioscapulohumeral Muscular Dystrophy;Statland;Continuum (Minneap. Minn.),2016
5. A unifying genetic model for facioscapulohumeral muscular dystrophy;Lemmers;Science,2010
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