Gene Therapy Grant Awarded, Drs. Steve Gray & Ben Philpot, UNC-CH

Gene therapy success stories are not only a hope but now becoming a reality. We won’t let Pitt Hopkins fall behind in this exciting and promising research. In August of 2016 the Pitt Hopkins Research Foundation awarded its first gene therapy grant, to Dr. Steve Gray at UNC and one of our pioneer heroes and researchers Dr. Ben Philpot. Here’s to Pitt Hopkins heroes old and new… And to the promise of gene therapy!

Grant summary

The laboratories of Dr. Ben Philpot and Dr. Steven Gray at the University of North Carolina at Chapel Hill are collaborating on a project to investigate the feasibility of a gene therapy approach for Pitt-Hopkins syndrome (PTHS). This collaborative study combines Dr. Philpot’s expertise in autism and neuroscience with Dr. Gray’s expertise in translational gene therapy for neurological disorders. The project will follow a platform gene transfer approach using AAV vectors taken by Dr. Gray to initiate a human Phase I trial for Giant Axonal Neuropathy. The approach uses an engineered virus, AAV, to carry a functional copy of the gene disrupted in PTHS into the body and distribute it across the nervous system. In this fashion, a single dose of this gene therapy could permanently restore the gene to cells across the nervous system, treating the disease at its source. This initial pilot study is meant to assess the potential of this as a treatment approach for PTHS, and identify any roadblocks that may exist.