Abstract
Duchenne muscular dystrophy (DMD) is a degenerative muscle disease caused by mutations in the DMD gene, which encodes the dystrophin protein. Recent advances in genome editing open doors for the development of potential “permanent” therapeutics. Today’s hottest genome editing tool, the CRISPR/Cas9 system, has recently been used to restore dystrophin reading frame and thus, its expression by deleting one or more exons encompassing the mutations in patient cells and in a mouse model of DMD. These approaches to restore dystrophin expression are highly promising, but many hurdles remain. This chapter summarizes the current state of genome editing therapeutic strategies for DMD and considerations for future development.
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