Article

CRISPR-induced deletion with SaCas9 restores dystrophin expression in dystrophic models <i>in vitro</i> and <i>in vivo</i>

2018-07-26

Abstract excerpt

Duchenne Muscular Dystrophy (DMD), a severe hereditary disease, affecting 1 boy out of 3500, mainly results from the deletion of one or more exons leading to a reading frame shift of the DMD gene that abrogates dystrophin protein synthesis. We used the Cas9 of Staphylococcus aureus (SaCas9) to edit the human DMD gene. Pairs of sgRNAs were meticulously chosen to induce a genomic deletion to not only restore the...

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Literature Corpus work: bd12f651-f188-56cf-9fde-20f0944d6115
DOI: 10.1101/378331

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