Regenerative Gene Therapy- Dr. Fumihiko Urano, MD, PhD

My current focus is to develop gene therapy for Wolfram syndrome. Our ultimate goal is to provide a cure using regenerative gene therapy. We have been trying to improve diabetes, visual acuity, and brain functions using viral vectors of a healthy Wolfram gene and a regenerative factor called MANF in mouse models. We are getting encouraging preliminary results and have published two articles recently. We are currently testing two ways to deliver genes through intravitreal (for optic nerve) and intraventricular (for brain) injections.