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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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49 items
fMRI of dog brain showing bright activation in the visual cortex.

Using fMRI, new vision study finds promising model for restoring cone function

Researchers have found that in dogs, fMRI can detect brain responses to daylight vision for black and white information as well as color information, and identify the area of the visual cortex that responds to stimulation of a cone-rich retinal region.
The image on the left is labeled "Normal retina," and shows green rods and red cones. The image on the right is labeled "Retina affected by retinitis pigmentosa," and shows green rods that have broken down.

Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice

By adapting virus-like particles to carry the machinery for a type of gene editing called prime editing, scientists have corrected disease-causing mutations in animals and increased editing efficiency.

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.

Precision eye therapy for dogs ready for human clinical development

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.

University of Houston researcher builds new model to examine Usher syndrome

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

Nanotechnology may improve gene therapy for blindness

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

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

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

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

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.