Gene Therapy Fellowship

Gene Therapy Fellowship

The Gene Therapy Fellowship at Nationwide Children’s provides a unique training opportunity for translational research in neuromuscular disease.

Genetic therapeutics for diseases did not exist a decade ago. Today, potential molecular therapies are entering clinical trials. The Center for Gene Therapy at Nationwide Children's 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 is unique in North America for its role in the translational development of gene therapies, especially for muscular dystrophies, motor neuron diseases and neurodegenerative diseases. The neuromuscular clinics at Nationwide Children’s in particular draw patients not only from across Ohio but across the country and internationally, both for patient care and to participate in trials. Gene Therapy fellows take an active role in the delivery of commercially available gene therapies and in the performance of ongoing research.

Trials conducted at Nationwide Children’s led to approvals of antisense exon skipping therapy for Duchenne muscular dystrophy and AAV-mediated gene replacement for spinal muscular atrophy. Current and recent DMD trials include studies using AAV to deliver microdystrophin as a gene replacement, U7snRNA-mediated exon skipping and delivery of GALGT2 as a surrogate gene therapy. Additional upcoming or ongoing gene transfer trials include AAV gene transfer studies in limb-girdle muscular dystrophies, SMARD1 and a variety of neurodegenerative disorders.

These cutting-edge trials are unique and offer an unparalleled training experience.