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First NIH-funded personalized drug development center in US will focus on muscle disease
Date:10/5/2011

ch at Children's National Medical Center and the CORT's co-principal investigator. "Patients with DMD have different mutations on various exons, so finding a treatment that is effective for the majority of patients involves looking at multiple defects simultaneously, which is what the Center aims to do."

Pursuit of AO drug development extends more than 20 years, particularly focused in cancer and inflammatory disease, although success in clinical trials has been limited. "We are seeing this type of therapeutic approach working better in DMD than other indications because of improved drug delivery, and more effective molecular targeting," noted Paula Clemens, MD, associate professor of neurology at the University of Pittsburgh and co-principal investigator of the new center. Dr. Qi Lu, director of McColl Lockwood Laboratory at Carolinas Medical Center and co-investigator of the CORT, provided additional rationale for improved efficacy in DMD: "We may need to reach only 20 percent of RNA targets in a successful 'rescue' application such as in DMD, whereas the previous 'knock-down' approaches in cancer and immunological disorders had to hit 90 percent of molecular targets." Dr. Lu's research in the new CORT will help optimize drugs to achieve the highest therapeutic potential for the majority of applicable DMD patients.

Research projects sponsored by the Center will include the first natural history study of Becker muscular dystrophy, a milder form of disease that is caused by mutations in the dystrophin gene. By studying specific mutations that cause Becker muscular dystrophy, CORT researchers will gain insights into the therapeutic potential of exon-skipping approaches. A second project will look at the biochemical function of the dystrophin proteins produced through application of the different exon-skipping dru
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Contact: Emily Hartman
ehartman@childrensnational.org
202-476-4500
Children's National Medical Center
Source:Eurekalert

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