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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Dettagli Bibliografici
Pubblicato in:Mol Ther Nucleic Acids
Autori principali: Rashnonejad, Afrooz, Amini-Chermahini, Gholamhossein, Taylor, Noah K., Wein, Nicolas, Harper, Scott Q.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2020
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7807095/
https://ncbi.nlm.nih.gov/pubmed/33510937
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2020.12.004
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