Treating Muscular Dystrophy with CRISPR Gene Editing
Type
Blog Post
...repair or add in disease-causing mutations, but, until recently it hadn’t been used to treat disease postnatally...the amount of research conducted on DMD, there is still no good treatment.
The dystrophin gene is very large...approach. Since shorter forms of dystrophin can still be functional, exon skipping is a good option for...CRISPR therapy closer to the clinic, much work is still needed. First, CRISPR delivery must be optimized...