Gene therapy successful in LCA trial

Jerusalem, Israel-Researchers in Israel have completed a clinical trial that successfully tested the use of gene therapy to restore sight to patients suffering from Leber's congenital amaurosis (LCA).

LCA, an inherited retinal dystrophy that causes congenital blindness, is caused by a mutation in the RPE65 gene. In this clinical trial, a normal RPE65 gene was injected into the retinas of two participants to replace the damaged gene and renew protein production.

Each participant was treated with the gene therapy in just part of the retina of one eye. Shortly after treatment, both participants noted a substantial improvement in their vision. The participants’ self-reporting of visual improvement was corroborated by objective, quantitative measurements of the treated area, according to the researchers.

“You cannot imagine what an effect this has had not only on the treated patients, their families, and on us, but also on the wider population of patients with retinal and macular degenerations here in Israel, who suddenly feel some glimmer of hope,” said researcher Eyal Banin, MD, PhD, of the Center for Retinal and Macular Degeneration at Hadassah University Medical Center, Jerusalem.

To watch a video with more information about this clinical trial, visit www.mvrf.org/news.php.

The research was funded by the Macula Vision Research Foundation (MVRF).

“We are extremely excited about the results of this study and feel confident that, in close collaboration with our partners across the globe, we are closer than ever to a cure for retinal diseases,” said Keith A. Lampman, executive director of the foundation. “MVRF is proud to be supporting the important work of Dr. Banin and others and look forward to continuing success in the future.”

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