Cedars-Sinai receives National Eye Institute grant to develop gene therapy for corneal disease

Cedars-Sinai stem cell researchers investigating ways to prevent eye problems in diabetic patients have been awarded a $3 million grant from the National Eye Institute to develop gene therapy in corneal stem cells to alleviate damage to corneas that can cause vision loss.

"Diabetes is the leading cause of blindness among working-age adults," said Alexander V. Ljubimov, PhD, director of the Ophthalmology Research Laboratories at the Cedars-Sinai Regenerative Medicine Institute and principal investigator on the five-year grant. "As more and more people are being diagnosed with diabetes, we are looking for ways to prevent some of the serious health conditions diabetes causes, such as blindness."

Although diabetes can damage the eye's retina and eventually cause blindness, the disease also strikes the cornea, the clear, dome-shaped surface that covers the front of the eye. Composed of cells and proteins, the cornea protects the rest of the eye from foreign objects, such as germs and dust. It also functions as a lens that allows light to pass to the back of the eye enabling the retina to perceive images.

To see well, all layers of the cornea must be free of any cloudy or opaque areas.

"In healthy patients, the cornea stays healthy and quickly heals possible defects because this tissue has stem cells that are constantly turning over and regenerating," said Clive Svendsen, PhD, director of the Cedars-Sinai Regenerative Medicine Institute and a co-investigator on the grant. "But in diabetic patients, something happens to the stem cells in the cornea. These cells become dysfunctional, and, because the corneal tissue doesn't turn over as occurs in healthy individuals, the diabetic cornea becomes prone to poor wound healing and other disorders that can cause pain and may lead to vision loss."

That condition is called diabetic keratopathy. Cedars-Sinai researchers recently showed for the first time that diabetics' corneal stem cells become abnormal because their corneas may stop producing certain proteins that enable normal functioning of corneal stem cells.

By employing gene therapy, researchers aim to correct production of abnormal proteins in diabetics' corneas in the hope that this treatment will restore stem cell functions, spurring normal corneal cell turnover and keeping the tissue healthy and transparent.

"This grant underscores Cedars-Sinai's deep commitment to fighting the growing diabetes epidemic in the U.S. by integrating our expertise in new frontiers in medicine: gene therapy and stem cell science," said Shlomo Melmed, MD, senior vice president of Academic Affairs, dean of the Cedars-Sinai Medical Faculty and the Helene A. and Philip E. Hixon Chair in Investigative Medicine. "We are honored that the National Eye Institute is supporting this critical research."