Treating Muscular Dystrophy with CRISPR Gene Editing
Type
Blog Post
...persist over time, stem cell editing would be highly desirable. Should stem cell editing occur, the CRISPR... high percentage of muscle cells throughout the body, especially stem cells
2. Remove any immunogenicity...patient myoblasts; when injected into DMD mice, these cells expressed functional dystrophin.
Editing genes in...in vivo is of course much more difficult than in cell culture, but Ran et al. have shown that CRISPR and...be delivered to both cardiac and skeletal muscle cells, where precision editing of the dystrophin gene ... the percentage of dystrophin-expressing muscle cells increases over time, and all three groups confirm...editing. Tabebordbar et al. show that muscle stem cells are modified by this approach, increasing the possibility...