My strategy is to stop/delay the progression of Wolfram syndrome using oral medications and restore functions of retinal ganglion cells, brain cells, and insulin-producing cells by regenerative gene therapy. Our first target is vision. We have been trying to improve visual acuity using viral vectors expressing a healthy Wolfram gene (WFS1) and a regenerative factor called MANF in cell and rodent models of Wolfram syndrome. Our preliminary results are encouraging. Dr. Venu Gurrum has recently joined my lab to accelerate the progress of this project. He is an expert in gene therapy for inherited optic nerve atrophy. My goal is to start a gene therapy trial in the next 3-7 years.
Therapeutic development – Regenerative Gene Therapy for Optic Nerve Atrophy
