Statement: New MDA-Funded Genetic Therapy Technique Targets DMD

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MDA research grantee Charles Gersbach announces potentially game-changing advance in gene modification for boys and young men with DMD.

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This is another prime example of MDA's commitment to making urgently-needed progress for those affected by muscular dystrophy and related life-threatening diseases." - Grace Pavlath, Ph.D.

Muscular Dystrophy Association research grantee Charles Gersbach, Assistant Professor of Biomedical Engineering at Duke University, recently announced a potentially game-changing advance in gene modification for boys and young men with Duchenne muscular dystrophy (DMD). The results were published Feb. 18 in Nature Communications, and Gersbach will discuss their implications at MDA's 2015 Scientific Conference, to be held March 11-14 in Washington, D.C.

Background: Gersbach and team are investigating a new genetic therapeutic technique that has the potential to treat more than half of patients fighting DMD and could be developed as a permanent, one-time treatment. Known as CRISPR-Cas9 genome editing, the strategy targets a large area of the dystrophin gene (DNA) and is designed to cause production of shorter-than-normal, but still functional, dystrophin protein in muscle tissue. If successful in humans, it could prolong function and increase longevity.

Other promising experimental DMD treatments – such as the experimental drugs eteplirsen and drisapersen – are currently being tested in clinical trials, but because these other treatments target dystrophin RNA rather than DNA, they would not result in a permanent correction and would require repeated treatment over a patient’s lifetime.

Ever since MDA-supported researchers identified flaws in the dystrophin gene as the cause of DMD in 1986, the organization has been in the forefront of DMD research, while continuing its commitment to ongoing patient care. One in three DMD patients in the U.S. attends an MDA-supported clinic, and thanks to MDA-funded laboratory and clinical investigations, there are now at least eight experimental compounds in clinical trials for DMD.

Statement from Grace Pavlath, Ph.D., MDA Senior Vice President & Scientific Program Director: "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."

About MDA

The Muscular Dystrophy Association is the world’s leading nonprofit health agency dedicated to saving and improving the lives of people with muscle disease, including muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases. It does so by funding worldwide research to find treatments and cures; by providing comprehensive health care services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising and local engagement.

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Claire Orphan
Media Relations Manager
(312) 260-5928
corphan(at)mdausa(dot)org

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Claire Orphan
Muscular Dystrophy Association
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