STEM cells have helped dogs with muscular dystrophy to walk again. Doctors hope a similar approach in humans could lead to more complete improvement than the other leading contender for a cure – an RNA-blocking drug now in clinical trials.
Duchenne muscular dystrophy (DMD) strikes about one in 3500 children, almost always boys, and is usually fatal by the age of 30. It is caused by mutations in the gene for the muscle protein dystrophin. Without it, muscle contractions shear and kill muscle cells, and victims become progressively weaker, often dying when their breathing muscles fail.
Giulio Cossu and colleagues at…
