Welcome to the Penn Neurogenetics Therapy Center

          The mission of the Penn Neurogenetics Therapy Center (PNTC) is to catalyze the translation of genetic diagnosis into precision, gene-based therapeutics for people with neurogenetic disorders. This involves a team approach which allows us to provide a complete perspective, from making complex genetic diagnoses to seamless delivery of new genetic-based therapies as they become commercially available. The PNTC has a core team of experts including neurologists with basic science backgrounds and translational experience, genetic counselors, as well as clinical research supporting staff, including a director of operations, business manager, project managers, research nurses, coordinators and assistants.

         Additionally, the PNTC interacts with thought leaders at Penn, including biomedical scientists, engineers, interventional radiologists and neurosurgeons. We actively interact with industry both for the evaluation of preclinical data and for planning of translational efforts. We are engaged in several, early phase clinical trials of novel therapies, including gene delivery via viral vectors. By leveraging Penn’s expertise in gene therapy and our large clinical practices, we are becoming a world leader in bringing genetic therapies for neurological disorders to the bedside.