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

Mol Ther Methods Clin Dev. 2020-12; 
Charis L Himeda, Takako I Jones, Peter L Jones
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摘要

Facioscapulohumeral muscular dystrophy (FSHD) is caused by incomplete silencing of the disease locus, leading to pathogenic misexpression of in skeletal muscle. Previously, we showed that CRISPR inhibition could successfully target and repress in FSHD myocytes. However, an effective therapy will require both efficient delivery of therapeutic components to skeletal muscles and long-term repression of the disease locus. Thus, we re-engineered our platform to allow delivery of more potent epigenetic repressors. We designed an FSHD-optimized regulatory cassette to drive skeletal muscle-specific expression of dCas9 from fused to HP1α, HP1γ, the MeCP2 transcriptional repression domain, or the SUV39H1 SET domain... More

关键词

AAV, CRISPR inhibition, DUX4, FSHD, dCas9, epigenetic repression, facioscapulohumeral muscular dystrophy, gene regulation, gene therapy, muscular dystrophy