Treating Muscular Dystrophy with CRISPR Gene Editing
Type
Blog Post
Published
Jan. 26, 2016, 3:30 p.m.
...X-linked recessive disorder affecting approximately 1 in 5000 males. DMD is caused primarily by frameshift...course much more difficult than in cell culture, but Ran et al. have shown that CRISPR and AAV can be used... applicable to 80% of DMD patients.
Figure 1: Exon skipping strategies for Duchenne muscular dystrophy...needed. First, CRISPR delivery must be optimized to:
1. Reach a high percentage of muscle cells throughout...also be closer than we might expect.
References
1. Kaiser J. CRISPR helps heal mice with muscular dystrophy...Thakore PI, Moreb EA, Rivera RM, Madhavan S, Pan X, Ran FA, Yan WX, Asokan A, Zhang F, Duan D, Gersbach CA...neurogenetic diseases. Nucleic Acid Ther. 2014 Feb;24(1):69-86. PubMed PMID: 24506781. PubMed Central PMCID...