A combined stem cell and gene therapy has been used to successfully treat mice with muscular dystrophy, reports a paper published in Nature Communications this week. The study, which uses genetically corrected induced pluripotent stem (iPS) cells derived from sick mice, might represent an important step towards the treatment of muscular dystrophies.
Duchenne muscular dystrophy is a progressive and incurable neuromuscular disease caused by mutations in the dystrophin gene. Rita Perlingeiro and her team took fibroblasts from mice with a severe form of muscular dystrophy and converted them into iPS cells. They then corrected the genetic defect, differentiated the cells into muscle precursor cells, and transplanted them back into the muscle of the respective donor mice. They found that the genetically corrected cells formed functional muscle fibres and mice showed improvements in muscle strength.