Treating Muscular Dystrophy with CRISPR Gene Editing
Type
Blog Post
Published
Jan. 26, 2016, 3:30 p.m.
...approach may also be applicable to a number of rare genetic diseases caused by splicing defects, including...estimated that exon skipping therapies would be applicable to 80% of DMD patients.
Figure 1: Exon...from Osterout et al. (2015b) under a CC-BY 4.0 license.
Long et al., Nelson et al., and Tabebordbar...Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases. Nucleic Acid Ther. ...