New MDA-Supported Genetic Therapy for DMD May Work With Only One Session

Charles Gersbach, Muscular Dystrophy Association research grantee and Biomedical Engineering Professor at Duke University, has recently announced an enormous and potentially game-changing development in gene modification for young men and boys suffering from Duchenne muscular dystrophy (DMD). The results were published in Nature Communications in a paper titled “Multiplex CRISPR/​Cas9-based genome editing for correction of ​dystrophin mutations that cause Duchenne muscular dystrophy.” Gersbach will be discussing the results and its implications at MDA’s 2015 Scientific Conference, which will be held in Washington, D.C. on March 11-14.

Gersbach and his research group are assessing a novel genetic therapeutic approach that might have the potential to address more than 50 percent of the patients fighting DMD and could be advanced as a permanent single session treatment. This strategy is known as CRISPR-Cas9 genome editing and functions by targeting a large area of the dystrophin gene (DNA). This approach is designed to cause the production of shorter but functional dystrophin protein in muscle tissue. If this method reveals to be successful in humans, it could imply the prolongation of its function and ultimately increase longevity.

Other experimental DMD treatments that might be promising are now being tested in clinical trials such as eteplirsen and drisapersen, both experimental drugs. However, since these treatments target dystrophin RNA instead of DNA, it would make impossible a permanent correction and would still require multiple treatment sessions over a patient’s lifetime.

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Since 1986, researchers supported by the MDA identified flaws in the dystrophin gene and linked this fact with DMD. Since then, the MDA organization has been leading DMD research and has continued its commitment regarding patient care.

“MDA is proud to have supported Dr. Gersbach for the development of this game-changing advance. This is another prime example of our commitment to making urgently-needed progress for those affected by muscular dystrophy and related life-threatening diseases. While we have every hope that other drugs currently in late-stage development for DMD will prove to be safe and effective, it is important to support multiple strategies in the development pipeline as MDA continues to search for treatments and cures,” stated Grace Pavlath.