AN RNA “bandage” that masks genetic mutations has prompted boys with Duchenne muscular dystrophy (DMD) to make a missing, muscle-strengthening protein throughout their bodies for the first time.
Around 1 in 3500 boys are born with DMD, the result of mutations in a gene on the X chromosome for the protein dystrophin. Boys with DMD tend to need wheelchairs by age 12 and die of cardiac or respiratory failure before they reach 30.
“When the dystrophin gene was identified 24 years ago, there were very high hopes that gene therapy would correct the condition,” says of the…
