Science. 2014 Sep 5;345(6201):1184-8. doi: 10.1126/science.1254445. Epub 2014 Aug 14.Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA.Long C, McAnally JR, Shelton JM, Mireault AA, Bassel-Duby R, Olson EN.
Muscular dystrophies fall in the groups of disorders that may be particularly amenable to gene therapy, as restoring gene products to even a fraction of deficient myofibers within a muscle may provide some improvement in overall muscle function.
Somewhat wistfully, this reminded me of the first transgenic "rescue" of the Mdx mouse by Greg Cox et al. in Jeff Chamberlain's lab at Michigan, back in 1993…
Nature. 1993 Aug 19;364(6439):725-9.Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity.Cox GA, Cole NM, Matsumura K, Phelps SF, Hauschka SD, Campbell KP, Faulkner JA, Chamberlain JS.