Treating Muscular Dystrophy with CRISPR Gene Editing
Type
Blog Post
...multiple exons that, when deleted through NHEJ, will restore the dystrophin reading frame. gRNAs bind in intronic... blot, at <10% of the wild-type level. Muscle histology is improved, with vastly reduced inflammation ...this result, Nelson et al. find that dystrophin restoration is maintained for at least six months.
Future..., Olson EN. Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular...