CRISPR mediated targeting of DUX4 distal regulatory element represses DUX4 target genes dysregulated in Facioscapulohumeral muscular dystrophy

Facioscapulohumeral muscular dystrophy (FSHD) is a debilitating muscle disease that currently does not have an effective cure or therapy. The abnormal reactivation of DUX4, an embryonic gene that is epigenetically silenced in somatic tissues, is causal to FSHD. Disease-specific reactivation of DUX4...

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Detalles Bibliográficos
Autores principales: Das, Sunny, Chadwick, Brian P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8206090/
https://www.ncbi.nlm.nih.gov/pubmed/34131248
http://dx.doi.org/10.1038/s41598-021-92096-0

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8206090/
https://www.ncbi.nlm.nih.gov/pubmed/34131248
http://dx.doi.org/10.1038/s41598-021-92096-0

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