NEI Research News

New CRISPR gene editing approach may also be useful for treating other inherited diseases, such as cystic fibrosis and familial hypercholesterolemia

UConn scientists led by Ephraim Trakhtenberg regrew optic nerves in mice through eye injections of fibronectin peptides (pieces of the larger protein).

National Eye Institute-funded scientists using gene editing corrected a mutation in a mouse model of retinitis pigmentosa, an inherited blinding eye disorder. The gene editing strategy restored production of rhodopsin, and retinal function and structure

About 79% of clinical trial participants experienced measurable improvement after receiving experimental, CRISPR-based gene editing that is designed to fix a rare form of blindness.

Researchers who work with tiny drug carriers known as lipid nanoparticles have developed a new type of material capable of reaching the lungs and the eyes, an important step toward genetic therapy for hereditary conditions like inherited vision loss.

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.

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.

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.

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.

Syndrome is a leading cause of combined deafness-blindness