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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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FDA-approved drug shows promise in lab models for blinding childhood disease 

March 29, 2023

A National Eye Institute-led team has identified a compound already approved by the U.S. Food and Drug Administration that keeps light-sensitive photoreceptors alive in three models of Leber congenital amaurosis type 10 (LCA 10).
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

Eye-opening discovery about adult brain’s ability to recover vision

October 5, 2022

University of California, Irvine team demonstrates the adult brain has the potential to partially recover from inherited blindness.
Retinal organoid with green photoreceptor outer segments.

NIH researchers develop gene therapy for rare ciliopathy

September 8, 2022

Researchers from the National Eye Institute (NEI) have developed a gene therapy that rescues cilia defects in retinal cells affected by a type of Leber congenital amaurosis (LCA), a disease that causes blindness in early childhood.

New UCI-led study lays foundation for establishing base editing as a one-time, durable treatment for inherited retinal degeneration

April 5, 2022

A new study indicates base editing may provide long-lasting retinal protection and prevent vision deterioration in patients with inherited retinal degeneration, specifically in Leber congenital amaurosis (LCA) patients.
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
Grantee News

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...

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