Article
Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.
Nature communications - 14 Feb 2017
Bengtsson Niclas E, Hall John K, Odom Guy L, Phelps Michael P, Andrus Colin R, Hawkins R David, Hauschka Stephen D, Chamberlain Joel R, Chamberlain Jeffrey S
Abstract excerpt
Gene replacement therapies utilizing adeno-associated viral (AAV) vectors hold great promise for treating Duchenne muscular dystrophy (DMD). A related approach uses AAV vectors to edit specific regions of the DMD gene using CRISPR/Cas9. Here we develop multiple approaches for editing the mutation in dystrophic mdx4cv mice using single and dual AAV vector delivery of a muscle-specific Cas9 cassette together with...
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