Duchenne muscular dystrophy is a devastating condition that destroys muscles because of a damaged gene for the protein dystrophin. Now, a new “molecular scalpel” shows promise in removing the damage and restoring dystrophin production to normal levels, the BBC reports.
The drug trial involved 19 children who were operated on using the scalpel, showing “real hope for the future,” according to the charity Muscular Dystrophy Campaign. Researchers injected tailored pieces of antisense RNA into the genetic code, which removed the damaged piece and allowed it to be matched up on either side of the mutation.
According to the BBC, the result is a shorter—but still functional—dystrophin.
At the end of the 12-week trial, seven of the 19 children had some degree of dystrophin production restored.
“The best result was 20 percent of normal dystrophin levels,” reported lead researcher Francesco Muntoni. “That is quite remarkable considering the study was for 12 weeks. I’ve worked with patients with Duchenne muscular dystrophy for many years and this is the first time we can say with confidence that we’ve made a significant breakthrough towards finding a targeted treatment.”
