Gene Editing: Hope for Muscular Dystrophy
London and Dallas Research Team
For the first time, there is real hope for a potential cure for Muscular Dystrophy. A team of scientists from the Royal Veterinary College in London and UT Southwestern Medical College in Dallas used gene therapy on dogs with the disease. They repaired a gene mutation that triggers the fatal condition.
This is an important step in the process to edit DNA in people with the fatal disease. 20,000 children, mostly boys, are diagnosed with it every year. Muscular Dystrophy is caused by a gene mutation that stops production of dystrophin, a protein that's essential to healthy muscle function. Without the protein muscles dramatically deteriorate.
The research was published in the journal Science. The scientists successfully used gene editing in 4 dogs with the disease. The procedure restored the production of dystrophin in the animals. Experts say much more research will be required before they can utilize it on humans.