Article

Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD

2022-01-25

Abstract excerpt

Duchenne muscular dystrophy (DMD) is the most prevalent inherited myopathy affecting children, caused by genetic loss of the gene encoding the dystrophin protein. There are currently four FDA-approved drugs for DMD that aim to restore expression of dystrophin by exon skipping using splice switching oligonucleotides. While these therapies require lifelong repeat administration, recent advancements in gene editing t...

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Literature Corpus work: 767aa6bd-4de1-50ad-aae1-86d5e9b7eaa1
DOI: 10.1101/2022.01.25.477678

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