Modified patient stem cells as prelude to autologous treatment of muscular dystrophy.
Davies KE., Grounds MD.
Duchenne muscular dystrophy is a devastating muscle wasting disease for which there is no effective treatment. In this issue of Cell Stem Cell, Benchaouir et al. (2007) demonstrate the delivery of genetically corrected CD133+ patient cells into mice, suggesting a new potential avenue for autologous cell therapy.