CRISPR Makes Strides Forward In Tackling Gene Mutation For Muscular Dystrophy In Beagles
While many companies pursue strategies to treat the disease caused by a lack of production of dystrophin, the gene-editing tool attempts to change the underlying cause. The scientists programmed the CRISPR system to cut the dogs’ DNA at a precise spot on the dystrophin gene. The cells repaired the cut, enabling dystrophin production to be restored.
The Wall Street Journal:
Crispr Used To Repair Gene Mutation In Dogs With Muscular Dystrophy
Researchers used a gene-editing tool to repair a gene mutation in dogs with Duchenne muscular dystrophy, an important step in efforts to someday use the tool to edit DNA in people with the same fatal disease. In a study published Thursday in the journal Science, researchers at UT Southwestern Medical Center in Dallas and the Royal Veterinary College in London reported that they used the Crispr gene-editing system in four dogs to restore production of dystrophin, a protein crucial for healthy muscle function. (Marcus, 8/30)
Stat:
CRISPR Fixed Duchenne In Dogs. Will This Tool Best An Older Gene Therapy?
It is the first published account of delivering CRISPR systemically (that is, throughout the body) in a large mammal. ...The UT study used up to 10 trillion adeno-associated viruses to ferry CRISPR into the muscles of two beagle puppies and into the bloodstream of two more; all the dogs had been born with a Duchenne-causing mutation very similar to one in people. After eight weeks, the beagle that received the highest dose of CRISPR had dystrophin levels in its muscle cells that were 25 percent to 70 percent of normal. Dystrophin levels in heart muscle reached 92 percent of normal. (Begley, 8/30)
