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Article

Methods of CRISPR/Cas9 Exon Skipping for Duchenne Muscular Dystrophy

2018-11-02

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive neuromuscular disease prevalent in 1 in 3500 to 5000 males worldwide. As a result of mutations that interrupt the reading frame of the dystrophin gene (DMD), DMD is characterized by a loss of dystrophin protein which leads to decreased muscle membrane integrity, which increases susceptibility to degeneration. CRISPR/Cas9 technology has garnered intere...

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Literature Corpus work
2604535a-0b24-54f8-af64-dd6db1a0fa26
DOI
10.20944/preprints201811.0018.v1
Open publication

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