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Sustained efficacy of CRISPR-Cas13b gene therapy for FSHD is challenged by immune response to Cas13b

2025-01-02

Abstract excerpt

Facioscapulohumeral muscular dystrophy (FSHD) is a potentially devastating muscle disease caused by de-repression of the toxic DUX4 gene in skeletal muscle. FSHD patients may benefit from DUX4 inhibition therapies, and although several experimental strategies to reduce DUX4 levels in skeletal muscle are being developed, no approved disease modifying therapies currently exist. We developed a CRISPR-Cas13b system...

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Literature Corpus work
add5e0f6-a1eb-5ea8-abea-c38bf1e8c2e7
DOI
10.1101/2024.12.18.629250
Open publication

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Sustained efficacy of CRISPR-Cas13b gene therapy for FSHD is challenged by immune response to Cas13bDOI 10.1101/2024.12.18.629250
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