Source
34 items

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.
Grey scale electron microscopy image of human retinal cells.

New genetic links found to rare eye disease, opening the door to better diagnostics and potential treatments

March 25, 2021

NEI-funded research at Scripps Research Institute has turned up more than a dozen gene variants linked to MacTel, a rare eye disease. The variants are likely causing the condition to develop and worsen for a significant share of patients.

New Research Sheds Light on Vision Loss in Batten Disease

February 5, 2021

New research shows how the mutation associated with Batten disease could potentially lead to degeneration of light sensing photoreceptor cells in the retina, and subsequent vision loss.

Study in mice shows genes may be altered through drug repurposing

December 17, 2020

Researchers at the University of Illinois Chicago have published a study showing a promising approach to using drug repurposing to treat genetic diseases.
David Gamm in the laboratory

UW researchers devise approach to treat rare, incurable form of blindness

July 30, 2020

Scientists at the University of Wisconsin‒Madison have published a proof-of-concept method to correct an inherited form of macular degeneration that causes blindness, and that is currently untreatable.
Multicolor image of retina in cross-section

Scientists Use Nanoparticle-Delivered Gene Therapy to Inhibit Blinding Eye Disease in Rodents

July 7, 2020

In experiments in rats and mice, two Johns Hopkins scientists report the successful use of nanoparticles to deliver gene therapy for blinding eye disease.
Microscopy image of lens and zone fibers stained red, blue and green.

New research on Marfan syndrome focuses on eyes

June 12, 2020

New NEI-supported research provides insight into the eye conditions associated with Marfan syndrome, where weakened zonule fibers cause vision problems.
Surface-dwelling fish with an eye, and pale cave fish without eye

Gene Found that Causes Eyes to Wither in Cavefish

June 2, 2020

University of Maryland-led study identified the first gene responsible for eye loss in cavefish, revealing connection to a human vascular disease.
Rhesus macaque monkey

Discovery in monkeys could lead to treatment for blindness causing syndrome

October 25, 2019

Oregon National Primate Research Center at OHSU reports first-ever nonhuman primate model for Bardet-Biedl Syndrome