MDA-supported researcher Charles Gersbach and colleagues have developed a strategy to change dystrophin DNA that has shown promise in the lab
MDA research grantee Charles Gersbach, assistant professor of biomedical engineering at Duke University, and colleagues, recently announced an advance in gene modification that could turn out to be a game-changer for boys and young men with Duchenne muscular dystrophy (DMD). The team's results were published Feb. 18, 2015, 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.
Potential for a permanent fix in 60 percent of DMD population
Gersbach is developing a new strategy called genome editing to modify flawed instructions in the dystrophingene. Dystrophin gene editing, which has so far shown promise in the laboratory in cultured cells and in mice, is designed to target and remove a large section of DNA from the dystrophin gene – an approach that could be beneficial to some 60 percent of DMD patients and could be developed as a permanent, one-time treatment. Known more specifically as CRISPR-Cas9 genome editing, the strategy 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.
Several drugs that are now being tested in late-stage clinical trials for DMD, such as eteplirsen and drisapersen, target dystrophin RNA rather than DNA, which would not bring about a permanent correction and would presumably need to be given repeatedly over a patient's lifetime.
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