American researchers have developed an approach to genomic editing of the DMD gene targeting muscle stem cells using the CRISPR / Cas 9 system associated with an AAV vector.
- When the product is injected into mdx mice with Duchenne muscular dystrophy, the researchers showed the existence of muscle fibers that produced dystrophin.
- mdx mice receiving muscle stem cells taken from mice treated with this method also exhibit dystrophin-producing muscle fibers.
- This production of dystrophin is maintained after several cycles of degeneration and regeneration.
These results confirm that it is possible to perform genomic editing in vivo on muscle stem cells to produce a stock of stem cells capable of repairing damaged muscle fibers with cells producing dystrophin and capable of self-renewal, making effective benefits over time.
