A new, NIH-supported translational research center will focus on exon skipping as a treatment for MD
A Center for Research Translation of Systemic Exon Skipping in Muscular Dystrophy has been established by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), part of the U.S. National Institutes of Health.
It will be headed by longtime MDA research grantee Eric Hoffman at the Children's Research Institute in Washington and will focus on a strategy called exon skipping to restore production of functional dystrophin protein in muscle tissue. Dystrophin is missing in Duchenne muscular dystrophy (DMD) and is only partially functional in Becker muscular dystrohpy (BMD).
In addition, the center's investigators plan to implement a study of the "natural history" (usual course) of BMD.
"The concept of the CORT [Center of Research Translation] is two-way," said NIAMS director Stephen Katz. "The Centers embrace both the translation of scientific discoveries toward clinical application, and clinical findings back to new research."
NIAMS announced the establishment of this new center Sept. 19, 2011.