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15 items
Image shows cone cells, labeled in red.

Turning back the clock on a severe vision disorder

March 31, 2021

Scientists at the University of Pennsylvania School of Veterinary Medicine report findings of a gene therapy to treat a severe form of Leber congenital amaurosis, caused by mutations in the NPHP5 gene.

Researchers use patients’ cells to test gene therapy for rare eye disease

January 28, 2021

Scientists at the National Eye Institute (NEI) have developed a promising gene therapy strategy for a rare disease that causes severe vision loss in childhood.

LSU Health New Orleans Discovers Drug Development Target for Retinal Dystrophies

December 1, 2020

A new study shows that deleting one of the inhibitors of the RPE65 gene in a mouse model that carries a human disease mutation prevents degeneration of cone photoreceptors that are used for daytime high-resolution color vision.

UCI-led study reveals significant restoration of retinal and visual function following gene therapy

October 19, 2020

New generation CRISPR technology lays foundation for therapeutics to treat a wide range of inherited ocular diseases
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Success of gene therapy for a form of inherited blindness depends on timing

September 9, 2019

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

December 4, 2018

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

September 4, 2018

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

August 4, 2015

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

May 3, 2015

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.
NEI logo

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

October 24, 2009

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.