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NEI Research News

Thanks to the work of NEI scientists and grantees, we’re constantly learning new information about the causes and treatment of vision disorders. Get the latest updates about their work — along with other news about NEI.

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Grantee News

Success of gene therapy for a form of inherited blindness depends on timing

Using a canine model of the vision disorder Leber congenital amaurosis, Penn researchers found that photoreceptor cells continue to deteriorate after treatment if it is given too late.
Multiphoton images show the outer segments of cone and rod photoreceptor cells in 215-day retinal organoids. Immunostaining shows a marker for cones (green) and rods (red). The “A” view shows optical sections; “B” is a reconstructed three-dimensional view.

NEI Awards Prize for Progress Toward Developing Lab-Made Retinas

The National Eye Institute (NEI) awarded $25,000 to a team led by Wei Liu, Ph.D., Albert Einstein College of Medicine, for demonstrating progress toward the development of a living model of the human retina...
Photo shows T. Michael Redmond, Ph.D.

NIH vision researcher T. Michael Redmond recognized with Champalimaud Vision Award

Vision researcher, T. Michael Redmond, Ph.D., chief of the National Eye Institute (NEI) Laboratory of Retinal Cell and Molecular Biology, is a recipient of the 2018 António Champalimaud Vision Award for foundational science discoveries about the molecular
Images of the brain generated by diffusion tensor imaging, a type of MRI. The red highlights show visual pathways in the brain that deteriorate in patients with LCA, but appear to improve with gene therapy to the retina. Credit: Dr. Manzar Ashtari, University of Pennsylvania.

With LCA Gene Therapy, a Rare Glimpse of the Adult Brain Adapting to New Experience

When people lose the ability to see, how do the visual parts of the brain change in response? And if they regain their sight, are the changes reversed?
LCA is an inherited disorder that causes vision loss in childhood. It primarily affects the functioning of the retina, the light-sensitive tissue at the back of the eye, as shown here. Photo credit: National Eye Institute

NIH-funded Study Points Way Forward for Retinal Disease Gene Therapy

Gene therapy for Leber congenital amaurosis (LCA), an inherited disorder that causes vision loss starting in childhood, improved patients’ eyesight and the sensitivity of the retina within weeks of treatment.
National Eye Institute logo.

New Findings Suggest Need for Combined Strategy in Treatment of Rare Form of Blindness

In 2008, a team of scientists funded by the National Eye Institute (NEI), a part of the National Institutes of Health, reported major progress in the treatment of an inherited form of progressive blindness using gene therapy.
National Eye Institute logo.

NEI Investment in RPE65-LCA Research Continues to Produce Promising Results

NEI support leading to gene therapy clinical trials for people with Leber congenital amaurosis (LCA) consisted of significant efforts by investigators in the intramural laboratories at the NEI.
National Eye Institute logo.

Promising Results in Phase 1 Gene Therapy Trial for Blinding Disease

Three young adults with an inherited form of blindness showed evidence of improved day and night vision following a specialized gene transfer procedure in a phase 1 clinical trial funded by the National Eye Institute (NEI), part of the NIH.
National Eye Institute logo.

Gene Therapy One Year Later: Patients Healthy and Maintain Early Visual Improvement

Three young adults who received gene therapy for a blinding eye condition remained healthy and maintained previous visual gains one year later, according to an August online report in Human Gene Therapy.
National Eye Institute logo.

Phase 1 Clinical Trial Results of Gene Transfer for Leber Congenital Amaurosis Reported

Three young adults with Leber Congenital Amaurosis-a severe degenerative disease of the retina caused by a mutation in the RPE65 gene-reported improvements in vision after undergoing a specialized gene transfer procedure.

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