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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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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...
Grantee News

Patient-Specific Stem Cells and Personalized Gene Therapy

Columbia University Medical Center (CUMC) researchers have created a way to develop personalized gene therapies for patients with retinitis pigmentosa (RP), a leading cause of vision loss.
Group of 5 scientists in a laboratory.

Mechanism that allows bacteria to infect plants may inspire cure for eye disease

By borrowing a tool from bacteria that infect plants, scientists have developed a new approach to eliminate mutated DNA inside mitochondria—the energy factories within cells.
National Eye Institute logo.

New Findings Suggest Need for Combined Strategy in Treatment of Rare Form of Blindness

In 2008, a team of scientists funded by the National Eye Institute (NEI), a part of the National Institutes of Health, reported major progress in the treatment of an inherited form of progressive blindness using gene therapy.
National Eye Institute logo.

NEI Investment in RPE65-LCA Research Continues to Produce Promising Results

NEI support leading to gene therapy clinical trials for people with Leber congenital amaurosis (LCA) consisted of significant efforts by investigators in the intramural laboratories at the NEI.
National Eye Institute logo.

Promising Results in Phase 1 Gene Therapy Trial for Blinding Disease

Three young adults with an inherited form of blindness showed evidence of improved day and night vision following a specialized gene transfer procedure in a phase 1 clinical trial funded by the National Eye Institute (NEI), part of the NIH.
National Eye Institute logo.

Gene Therapy One Year Later: Patients Healthy and Maintain Early Visual Improvement

Three young adults who received gene therapy for a blinding eye condition remained healthy and maintained previous visual gains one year later, according to an August online report in Human Gene Therapy.

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