Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes

Facioscapulohumeral muscular dystrophy (FSHD) arises from epigenetic changes that de-repress the DUX4 gene in muscle. The full-length DUX4 protein causes cell death and muscle toxicity, and therefore we hypothesize that FSHD therapies should center on inhibiting full-length DUX4 expression. In this...

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Detalles Bibliográficos
Autores principales: Rashnonejad, Afrooz, Amini-Chermahini, Gholamhossein, Taylor, Noah K., Wein, Nicolas, Harper, Scott Q.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7807095/
https://www.ncbi.nlm.nih.gov/pubmed/33510937
http://dx.doi.org/10.1016/j.omtn.2020.12.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7807095/
https://www.ncbi.nlm.nih.gov/pubmed/33510937
http://dx.doi.org/10.1016/j.omtn.2020.12.004

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