Targeted epigenetic repression by CRISPR/dSaCas9 suppresses pathogenic DUX4-fl expression in FSHD

Facioscapulohumeral muscular dystrophy (FSHD) is caused by incomplete silencing of the disease locus, leading to pathogenic misexpression of DUX4 in skeletal muscle. Previously, we showed that CRISPR inhibition could successfully target and repress DUX4 in FSHD myocytes. However, an effective therap...

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Detalles Bibliográficos
Autores principales: Himeda, Charis L., Jones, Takako I., Jones, Peter L.
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/PMC7806950/
https://www.ncbi.nlm.nih.gov/pubmed/33511244
http://dx.doi.org/10.1016/j.omtm.2020.12.001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7806950/
https://www.ncbi.nlm.nih.gov/pubmed/33511244
http://dx.doi.org/10.1016/j.omtm.2020.12.001

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