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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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41 items
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.
Samarendra Mohanty and Subrata Batabyal

Scientists use gene therapy and a novel light-sensing protein to restore vision in mice

October 22, 2020

A newly developed light-sensing protein called the MCO1 opsin restores vision in blind mice when attached to retina bipolar cells using gene therapy.

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