Longitudinal measures of RNA expression and disease activity in FSHD muscle biopsies

Advances in understanding the pathophysiology of facioscapulohumeral dystrophy (FSHD) have led to the discovery of candidate therapeutics, and it is important to identify markers of disease activity to inform clinical trial design. For drugs that inhibit DUX4 expression, measuring DUX4 or DUX4-targe...

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Detalhes bibliográficos
Publicado no:Hum Mol Genet
Main Authors: Wong, Chao-Jen, Wang, Leo H, Friedman, Seth D, Shaw, Dennis, Campbell, Amy E, Budech, Chris B, Lewis, Leann M, Lemmers, Richard J F L, Statland, Jeffrey M, van der Maarel, Silvère M, Tawil, Rabi N, Tapscott, Stephen J
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2020
Assuntos:
General Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7158378/
https://ncbi.nlm.nih.gov/pubmed/32083293
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddaa031
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