Research to Prevent Blindness

Gene Therapy Proven Effective for Patients with Inherited Retinal Disease

Dr. Jean Bennett

Jean Bennett, M.D., Ph.D., of the University of Pennsylvania, has established the scientific underpinnings which made it possible to test the first potential definitive retinal gene therapy treatment for patients with blinding retinal degenerations.

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.

Related News: Retinitis Pigmentosa

clock icon

Researchers Build An “Aging Clock” To Help Treat Eye Disease

The new way to measure ocular aging opens treatment avenues for numerous eye diseases.

Read More

RPB logo

Research to Prevent Blindness Marks $400 Million in Funding to Advance Eye Disease Research

RPB funds a new round of researchers and hits a milestone in supporting vision-related breakthroughs.

Read More

Retina of a person with retinitis pigmentosa

Understanding Usher Syndrome: New Findings

RPB-supported researchers make progress in characterizing the genetic components of Usher syndrome.

Read More


Edited Stem Cells Offer Hope for RP Therapy

Findings raise the possibility of treating blinding eye diseases using a patient's own corrected cells as replacement tissue

Read More

Dr. Tsang

Renewed Hope for Gene Therapy to Restore Vision

Findings indicate that retinitis pigmentosa is treatable even at advanced disease stages.

Read More

Dr. Gamm stem cell researcher
Watch The Video

RPB Accelerates Retinitis Pigmentosa Research with Awards to Leading Scientists

RPB is providing $500,000 to accelerate the development of treatments for Retinitis Pigmentosa (RP) -- a family of retinal diseases that progressively create extreme tunnel vision, loss of night vision and leave...

Read More



Get our email updates filled with the latest news from our researchers about preventing vision loss, treating eye disease and even restoring sight. Unsubscribe at any time. Under our privacy policy, we'll never share your contact information with a third party.