Stammzelltherapien für Muskeldystrophien

Muscular dystrophies are devastating and untreatable genetic diseases leading to progressive muscle degeneration and weakness. The expanding landscape of CRISPR-Cas-based genome editing tools allows the in situ repair of many disease-causing mutations in patient cells in an unprecedented manner. Here, I discuss recent advances and challenges for using gene edited muscle stem cells in autologous cell replacement therapies to treat muscular dystrophy.