Washington D.C: A new study has found that gene-editing technique can successfully put a halt to Duchenne muscular dystrophy progression.
In the research undertaken by Ut Southwestern Medical Center, scientists have found this technique could lead to one of the first successful genome editing-based treatments if efficiently and safely scaled up in DMD patients.
Researchers found that DMD, the most common and severe form of muscular dystrophy among boys, is characterised by progressive muscle degeneration and weakness. It is caused by mutations in the X-linked DMD gene that encodes the protein dystrophin.
The disease affects one in 3,500 to 5,000 boys, according to the Centers for Disease Control and Prevention and other estimates, and often leads to premature death by the early 30s.
This condition often results in heart muscle disease or cardiomyopathy, the leading cause of death in these patients.
Lead researcher and senior author Dr. Eric Olson said this is different from other therapeutic approaches because it eliminates the cause of the disease. To test this out, researchers delivered gene-editing components to the mice via adeno-associated virus 9 (AAV9). DMD mice treated with this technique produced dystrophin protein and progressively showed improved structure and function of skeletal muscle and heart.
The study is published in the Journal Science.