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BioCentury | Jul 30, 2021
Distillery Therapeutics
CRISPR-based FKRP correction for a broad spectrum of muscular dystrophies
DISEASE CATEGORY: Musculoskeletal
INDICATION: Muscular dystrophy Replacing the mutant fukutin-related protein (FKRP) gene with the wild-type sequence using CRISPR-Cas9 technology could treat
INDICATION: Muscular dystrophy Replacing the mutant fukutin-related protein (FKRP) gene with the wild-type sequence using CRISPR-Cas9 technology could treat
BioCentury | Jun 5, 2021
Targets & Mechanisms
Going beyond genetic medicines for muscular dystrophy
Muscular dystrophy programs in the clinic cover not only a range of muscle mechanisms but also anti-fibrotic and anti-inflammation ones
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