Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disease caused by mutations in the gene encoding dystrophin. Three papers now report the use of adeno-associated viruses to locally or systemically deliver the clustered regularly-interspaced short palindromic repeats (CRISPR)–Cas9 system to the mdx mouse model of DMD, to remove the mutated exon 23 from the dystrophin gene. This gene-editing approach resulted in expression of the modified dystrophin gene and partial restoration of dystrophin protein expression in skeletal and cardiac muscle, as well as in muscle stem cells, leading to improved skeletal muscle function in mdx mice.
References
Tabebordbar, M. et al. In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science 351, 407–411 (2016)
Nelson, C. E. et al. In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science 351, 403–407 (2016)
Long, C. et al. Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Science 351, 400–403 (2016)
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Crunkhorn, S. Gene editing restores muscle function. Nat Rev Drug Discov 15, 160 (2016). https://doi.org/10.1038/nrd.2016.36
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DOI: https://doi.org/10.1038/nrd.2016.36
