Very Promising Muscular Dystrophy Research

[samclem]

I saw a write-up today on gene editing techniques tested to treat Duchenne muscular dystrophy, the most common form of MD. I thought some here might be interested.
Here's the magazine article.

From another article (in part):

Scientists for the first time have used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in a large mammal, according to a study by UT Southwestern that provides a strong indication that a lifesaving treatment may be in the pipeline.


DMD, which affects one in 5,000 boys, leads to muscle and heart failure, and premature death by the early 30s. Patients are forced into wheelchairs as their muscles degenerate and eventually onto respirators as their diaphragms weaken. No effective treatment exists, though scientists have known for decades that a defect in the dystrophin gene causes the condition.
The latest research applied the same technique in four dogs that shared the type of mutation most commonly seen in DMD patients.

. . . .
CRISPR edited the exon, and within several weeks the missing protein was restored in muscle tissue throughout the body, including 92 percent correction in the heart and 58 percent in the diaphragm, the main muscle needed for breathing.

Interesting that this report should come out so close to Labor Day. As someone who remembers those Jerry Lewis MDA Labor Day telethons, it is great to see a glimmer of hope after all this time.