The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) of the National Institutes of Health announced four new Centers of Research Translation awards today. Nationwide Children’s Hospital researchers have been awarded a $7.5 million grant - $1.5 million per year for five years - from the National Institutes of Health to develop a Center for Research Translation designed to accelerate new therapies for muscular dystrophies into clinical practice.
“This funding allows us to optimize the use of the unique resources and broad range of expertise in the Center for Gene Therapy at Nationwide Children’s,” says Kevin Flanigan, MD, a neurologist and principal investigator in the center and project director for the grant.
The projects funded by the award will focus on congenital and limb-girdle muscular dystrophies, facioscapulohumeral muscular dystrophy (FSHD) and Duchenne muscular dystrophy, which are some of the most common forms of the neuromuscular disorder.
“Despite their differences, we believe the expertise involved in translating therapies for one form of muscular dystrophy can be applied to other forms,” notes Dr. Flanigan, who is also a professor of Pediatrics and Neurology at the Ohio State University College of Medicine. “For example, we plan to extend a current therapy designed for Duchenne muscular dystrophy to congenital and limb-girdle forms of the disorder as well as examine potential viral-based therapies for FSHD.”
The Center for Gene Therapy at Nationwide Children’s investigates and employs the use of gene and cell- based therapeutics for the prevention and treatment of human diseases, particularly focusing on neuromuscular and neurodegenerative diseases.