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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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Image of a mouse retina

NIH scientists deploy CRISPR to preserve photoreceptors in mice

Silencing a gene called Nrl in mice prevents the loss of cells from degenerative diseases of the retina, according to a new study. The findings could lead to novel therapies for preventing vision loss from human diseases such as retinitis pigmentosa.
Grantee News

Vitamin B3 Prevents Glaucoma, JAX Researchers Find

In mice genetically predisposed to glaucoma, vitamin B3 added to drinking water is effective at preventing the disease.
Grantee News

CRISPR Used to Repair Blindness-causing Genetic Defect in Patient-derived Stem Cells

Columbia University Medical Center (CUMC) and University of Iowa scientists have used a new gene-editing technology called CRISPR to repair a genetic mutation responsible for retinitis pigmentosa (RP), an inherited condition.
Grantee News

Gene Editing Technique Improves Vision in Rats with Inherited Blindness

A new technique that has the potential to treat inherited diseases by removing genetic defects has been shown for the first time to hinder retinitis pigmentosa, a degenerative eye disease with no known cure that can lead to blindness.
Grantee News

Researchers Grow Retinal Nerve Cells in the Lab

Johns Hopkins researchers have developed a method to efficiently turn human stem cells into retinal ganglion cells, the type of nerve cells located within the retina that transmit visual signals from the eye to the brain.
Image of gene therapy preserved vision in a study involving dogs.

Gene Therapy Staves Off Blindness from Retinitis Pigmentosa in Canine Model

Gene therapy preserved vision in a study involving dogs with naturally occurring, late-stage retinitis pigmentosa, according to research funded by the National Eye Institute (NEI), part of the National Institutes of Health.
Dr. John Guy and colleagues added a homing signal to a virus in order to deliver the ND4 gene into mitochondria. A marker for the gene is shown in red and the ND4 protein is shown in green, inside retinal ganglion cells in the mouse eye. The nuclei of retinal ganglion cells are shown in blue. Credit: Dr. Hong Yu, Bascom Palmer.

Scientists Test New Gene Therapy for Vision Loss From a Mitochondrial Disease

Researchers funded by the National Institutes of Health have developed a novel mouse model for the vision disorder Leber hereditary optic neuropathy (LHON), and found that they can use gene therapy to improve visual function in the mice.
Images of the brain generated by diffusion tensor imaging, a type of MRI. The red highlights show visual pathways in the brain that deteriorate in patients with LCA, but appear to improve with gene therapy to the retina. Credit: Dr. Manzar Ashtari, University of Pennsylvania.

With LCA Gene Therapy, a Rare Glimpse of the Adult Brain Adapting to New Experience

When people lose the ability to see, how do the visual parts of the brain change in response? And if they regain their sight, are the changes reversed?
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

NIH-funded Study Points Way Forward for Retinal Disease Gene Therapy

Gene therapy for Leber congenital amaurosis (LCA), an inherited disorder that causes vision loss starting in childhood, improved patients’ eyesight and the sensitivity of the retina within weeks of treatment.
Cross-sectional images of retina from retinoschisin-deficient mice, untreated and treated with XLRS gene therapy.

NEI Human Gene Therapy Trial for Retinoschisis Underway

The National Eye Institute (NEI) recently launched the first-ever human gene therapy trial for the vision disorder X-linked retinoschisis (XLRS). Researchers are conducting the trial at the National Institutes of Health Clinical Research Center in...