Article

Reversal of molecular pathology by RNA-targeting Cas9 in a myotonic dystrophy mouse model

2017-09-04

Abstract excerpt

The dominantly inherited, multi-systemic disease myotonic dystrophy type I (DM1) is caused by triplet repeat CTG expansions in the DMPK gene and is the most common form of adult-onset muscular dystrophy. Elimination of the toxic, repetitive CUG RNA constitutes a therapeutic for this disease. We report an RNA-targeting Cas9 (RCas9) system that supports efficient reversal of DM1 phenotypes via delivery to adult pol...

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Literature Corpus work: 15290a8a-2af4-5990-8cd2-733dae9bb6cc
DOI: 10.1101/184408

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