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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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23 items
Human iPSC-derived retinal pigment epithelium (RPE).

UC Irvine study shows similarities and differences in human and insect vision formation

Researchers have discovered profound similarities and surprising differences between humans and insects in the production of the critical light-absorbing molecule of the retina, 11-cis-retinal, also known as the “visual chromophore.”

New gene-editing technique holds potential for treating childhood blindness

Using a new experimental technique to fix faulty eye cells, a team led by University of Wisconsin researchers was able to repair a gene mutation that causes one form of childhood blindness.
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FDA-approved drug shows promise in lab models for blinding childhood disease 

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

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

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

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

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

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

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

New generation CRISPR technology lays foundation for therapeutics to treat a wide range of inherited ocular diseases

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