Treating Muscular Dystrophy with CRISPR Gene Editing
Type
Blog Post
Published
Jan. 26, 2016, 3:30 p.m.
...protein’s reading frame, leading to the production of a smaller, but at least partially functional protein...individual experiences progressive muscle wasting leading to death at around 30 years of age. Despite the...depositor Charles Gersbach used paired zinc finger nucleases to remove exon 51 in DMD patient myoblasts. They...force, and other muscle tests, muscle function is clearly improved, although not to wild-type levels. With...that dystrophin restoration is maintained for at least six months.
Future directions and obstacles to clinical...through genomic excision of exon 51 by zinc finger nucleases. Mol Ther. 2015a Mar;23(3):523-32. PubMed PMID...PMC4393360.
Additional Resources on the Addgene Blog
Learn How CRISPR Could Be Used to Treat Cataracts and ...