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

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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Bibliographic Details
Main Authors: Charis L. Himeda, Takako I. Jones, Peter L. Jones
Format: Article
Language:English
Published: Elsevier 2021-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
FSHD
facioscapulohumeral muscular dystrophy
DUX4
CRISPR inhibition
dCas9
gene therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120302497

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