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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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Precision eye therapy for dogs ready for human clinical development

April 28, 2023

A successful gene therapy trialed at Michigan State University in dogs with an inherited eye disease is ready to be developed for clinical use in human patients with a rare condition called retinitis pigmentosa.
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University of Houston researcher builds new model to examine Usher syndrome

April 18, 2023

Syndrome is a leading cause of combined deafness-blindness
3D illustration of nanoparticle inside an eye

Nanotechnology may improve gene therapy for blindness

January 11, 2023

Using nanotechnology that enabled mRNA-based COVID-19 vaccines, a new approach to gene therapy may improve how physicians treat inherited forms of blindness.

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.

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