Researchers at UT Southwestern Medical Center say they’ve developed an improved gene-editing technique to correct a common mutation that causes Duchenne muscular dystrophy.
When they used the new single-cut technique using CRISPR/Cas9 tools on a new mouse model they also developed to study the disease, the mice showed improved muscle strength, the researchers reported in Science Translational Medicine.
“We think these advancements will be valuable for the field, and can help us move closer to tackling this disease in humans,” Dr. Eric Olson, director of the Hamon Center for Regenerative Science and Medicine, said in a news release.
The new approach restored up to 90 percent of dystrophin protein expression throughout the skeletal muscles and the heart in the mouse model, the researchers said. The lack of dystrophin protein is what leads to muscle and heart failure, and eventually premature death, from Duchenne muscular dystrophy.
DMD is the most common—and the most severe—form of muscular dystrophy, causing muscle fibers to break down, and often leading to death in early childhood. It’s most prevalent in boys.
Olson’s team first used CRISPR/Cas9-mediated genome editing to correct the mutation in mice and prevent muscular dystrophy in 2014. Since then they’ve developed the techniques to successfully edit defective genes in mice that have the disease, as well as in human cells, and are working toward developing the techniques for eventual human trials.