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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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NEI researchers home in on a new cause of Stargardt disease

October 27, 2022

Using a new stem-cell based model made from skin cells, scientists found the first direct evidence that Stargardt-related ABCA4 gene mutations affect a layer of cells in the eye called the retinal pigment epithelium (RPE).
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.
Comparison of two retinal images.

Gene therapy for rare eye disease safe but lacks efficacy in early trial

May 31, 2022

A 28-patient phase 1 gene therapy clinical trial for the degenerative retinal disease Leber hereditary optic neuropathy (LHON) found no significant safety concerns; however, treatment failed to improve or slow vision loss, with even the highest dose.
Stained image of retina

Correcting night blindness in dogs

March 22, 2022

University of Pennsylvania researchers have developed a gene therapy that restores dim-light vision in dogs with a congenital form of night blindness, offering hope for treating a similar condition in people.

University of Oregon researchers develop gene therapy for eye disease

August 3, 2021

Researchers have developed a new gene therapy that could eventually provide an alternative treatment for Fuchs’ endothelial corneal dystrophy, a genetic eye disease affecting roughly one in 2,000 people globally.
Eye is shown connected to visual processing regions of the brain via the optic nerve. Enlargement of retina shows photorecetors, biopolar cells and retinal ganglion cells.

Scientists discover gene therapy provides neuroprotection to prevent glaucoma vision loss

July 22, 2021

A form of gene therapy protects optic nerve cells and preserves vision in mouse models of glaucoma, according to research supported by NIH’s National Eye Institute. The findings suggest a way forward for developing neuroprotective therapies for glaucoma.

Gene therapy shows promise in treating rare eye disease in mice

April 13, 2021

A gene therapy protects eye cells in mice with a rare disorder that causes vision loss, especially when used in combination with other gene therapies, shows a study published in eLife.
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.

A new vision for AAV-delivered gene therapies

February 10, 2021

A broadly applicable AAV genome-coupled immunomodulation strategy helps cloak the AAV virus from unwanted immune responses, and offers important insights into ocular inflammation.

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.