Gene Therapy Featured Research and Projects

Gene Therapy Research and Projects

Areas of Focus

  • Muscular dystrophies

  • Neuromuscular diseases

  • Peripheral neuropathies

  • Lysosomal storage disorders

  • Ischemia and perfusion injury

  • Cancer and regenerative medicine

  • AAV gene transfer vector development

  • Muscle stem cells


Featured Research and Projects

  • Nationwide Children’s Hospital and The Ohio State University Wexner Medical Center are part of a select group of 25 national clinical sites that make up the Network of Excellence in Neuroscience Clinical Trials (NeuroNEXT) program.

  • Phase I clinical trials of a rAAV based HIV-1 vaccine in collaboration with Children’s Hospital of Philadelphia, the International AIDS Vaccine Initiative, NIH (NIAID) and Targeted Genetics Corp.


  • Translational research of novel treatment strategies for muscular dystrophino-pathies using genes that correct or modify the disease phenotype (e.g. micro-dystrophin, galgt2, follistatin, exon-skip inducing RNA and RNAi).


  • Clinical and pre-clinical development of novel, neurotrophic-based treatment strategies for amyotrophic lateral sclerosis (ALS). Viral vector mediated gene transfer is being utilized to provide factors for neuronal growth and protection of affected motor neurons.


  • Research into second-generation gene transfer rAAV vectors targeting multiple organs, that allow for delivery through the vasculature.


  • Research in the isolation, growth and engraftment potential of muscle derived stem cells to treat a host of degenerative muscle diseases.


  • Pre-clinical studies for the treatment of the lysosomal storage disorder MPS IIIB using viral mediated gene transfer systemically or targeted to the central nervous system (CNS).


  • cGMP Viral Vector Manufacturing Facility to support Phase I and Phase II clinical trials.

Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000