Treating Muscular Dystrophy with CRISPR Gene Editing
Type
Blog Post
Published
Jan. 26, 2016, 3:30 p.m.
...disease postnatally. Now, three papers published concurrently in Science have shown CRISPR can treat a genetic... Even low-frequency editing could make a huge difference in DMD, and it’s estimated that exon skipping...respectively. Although each study used slightly different methods, they each observed the same positive ...necrosis compared to unedited animals. In grip strength, specific force, and other muscle tests, muscle...disease type C. Although the three DMD studies referenced here represent a great step forward for CRISPR...many distinct CRISPR therapies to match these different mutations. For each therapy, effectiveness and...treatments may also be closer than we might expect.
References
1. Kaiser J. CRISPR helps heal mice with muscular...