Treating Muscular Dystrophy with CRISPR Gene Editing
Type
Blog Post
...Becker muscular dystrophy (BMD).
Dystrophin’s size makes it difficult to deliver via gene therapy, so researchers...normal function. Even low-frequency editing could make a huge difference in DMD, and it’s estimated that...Gootenberg JS, Kriz AJ, Zetsche B, Shalem O, Wu X, Makarova KS, Koonin EV, Sharp PA, Zhang F. In vivo genome...