Neuromuscular Genetic Therapeutics Fellowship
The Nationwide Children's Hospital Fellowship in Neuromuscular Genetic Therapeutics (NGT) provides a unique training opportunity for translational research in neuromuscular disease.
Genetic therapeutics for neuromuscular diseases did not exist a decade ago, but today potential molecular therapies are entering clinical trials. The Center for Gene Therapy at Nationwide Children's Hospital is now a leading site for the translational development of these therapies. Our goal is to train clinician scientists to transform basic advances into the next generation of novel therapies for patients.
The Center for Gene Therapy at Nationwide Children's Hospital is unique in North America in translational development of therapies for muscular dystrophies. The Muscular Dystrophy Association clinic draws patients throughout Ohio and across the country for both patient care and to participate in trials. Current and recent trials include studies of exon-skipping and nonsense suppression genetic therapies. Upcoming or ongoing gene transfer trials include follistatin gene transfer in patients with Becker muscular dystrophy or inclusion body myositis; alpha-sarcoglycan gene transfer in patients with limb-girdle muscular dystrophy; micro-dystrophin gene transfer in patients with Duchenne muscular dystrophy; and NAGLU gene transfer in mucopolysaccharidosis type IIIB. These cutting edge trials are unique, and offer an unparalleled training experience.