Neuromuscular Genetic Therapeutics Fellowship

Kevin Flanigan, MD
Program Director
Phone: (614) 355.2947
Fax: (614) 722.3273
E-Mail: Kevin.Flanigan@NationwideChildrens.org

Choumpree Jenkins
Program Coordinator
Phone: (614) 722.5615
Fax: (614) 722.3273
E-Mail: Choumpree.Jenkins@NationwideChildrens.org

Fellowship Overview

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.  Many of these are first human trials of novel therapies.  Dr. Jerry Mendell led the first trials of gene transfer for both limb-girdle muscular dystrophy type 2D and Duchenne muscular dystrophy.  Dr. Kevin Flanigan began the first North American trials of antisense oligonucleotide induced exon skipping in patients with Duchenne muscular dystrophy (DMD) in July 2010, and Dr. Mendell began the second, using a different chemistry, in July 2011.  Trials of antisense exon skipping are already in planning over the next two years, in both ambulant and non-ambulant patients.  Funded gene transfer trials are mapped out to occur at Nationwide Children's over the next four years.  These include a trial of follistatin gene transfer in patients with Becker muscular dystrophy or inclusion body myositis.  Other therapeutic approaches for which pre-Investigational New Drug (IND) interactions are underway include 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.