Gene Therapy Proven Effective for Patients with Inherited Retinal Disease

Researchers have used gene therapy to safely restore vision in three young adults with a form of retinitis pigmentosa known as Leber's congenital amaurosis (LCA), an inherited retinal disorder that reduces vision in early childhood and progresses toward total blindness during a patient's 20s or 30s.  There is no currently available treatment for LCA . Read the article in The New England Journal of Medicine.  Read the University of Pennsylvania press release.

RPB grants, spanning 15 years, contribute to lead researcher's success
Jean Bennett, M.D., Ph.D., one of the lead researchers on the international team of scientists, has received grant support from RPB since 1992 in the form of four awards, all toward this breakthrough gene therapy approach.  Her husband, Albert M. Maguire, M.D., also from The University of Pennsylvania's Scheie Eye Institute and the lead surgeon on the clinical trial, has received a grant from RPB for this research as well.

Dr. Bennett's recent RPB Senior Scientific Investigator Award, in 2007, will help her develop and optimize gene therapy for a second, more prevalent form of LCA.  Strategies that emerge from that investigation may provide a blueprint that could be applied to a large number of other retinal diseases that initiate in photoreceptor cells.  She will be collaborating with Edward N. Pugh, Jr., Ph.D., recipient of the prestigious RPB Jules and Doris Stein Research Professorship.

April 28, 2008