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CRISPR/dCas9-mediated Transcriptional Inhibition Ameliorates the Epigenetic Dysregulation at D4Z4 and Represses DUX4-fl in FSH Muscular Dystrophy

Facioscapulohumeral muscular dystrophy (FSHD) is one of the most prevalent myopathies, affecting males and females of all ages. Both forms of the disease are linked by epigenetic derepression of the D4Z4 macrosatellite repeat array at chromosome 4q35, leading to aberrant expression of D4Z4-encoded R...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Himeda, Charis L, Jones, Takako I, Jones, Peter L
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4786914/
https://ncbi.nlm.nih.gov/pubmed/26527377
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.200
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