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

First National Institutes of Health-funded personalized drug development center in the U.S. will focus on muscle disease New Center of Research Translation awarded to Children's National Medical Center, University of Pittsburgh School of Medicine and Carolinas Medical Center to accelerate R&D

Washington, DC The first Center of Research Translation (CORT), focused on personalized drug development for Duchenne muscular dystrophy (DMD), has been created through a $7.9 million grant from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) of the National Institutes of Health. The 5-year grant was awarded to a consortium of academic laboratories at Children's National Medical Center in Washington, DC, University of Pittsburgh in Pittsburgh, PA, and Carolinas Medical Center in Charlotte, NC.

The Center's personalized drug development will focus on a series of small molecule drugs, called antisense oligonucleotides (AO), which target specific mutations in the dystrophin gene to partially correct the DMD genetic defect at the mRNA splicing level. This approach is called "exon skipping," and is intended to help DMD patients produce dystrophin, the protein that is missing in their muscles. Exons are those segments on a gene that contain the necessary genetic information to produce a protein.

"While there has been very promising clinical trial data recently reported for specific exon 51 AO drugs in DMD, the Center of Research Translation will lay the groundwork for the next series of exon-specific drugs," said Eric Hoffman, PhD, director of the Center for Genetic Medicine Resear
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Contact: Emily Hartman
ehartman@childrensnational.org
202-476-4500
Children's National Medical Center
Source:Eurekalert

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