NEI Research News

Using nanotechnology that enabled mRNA-based COVID-19 vaccines, a new approach to gene therapy may improve how physicians treat inherited forms of blindness.

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).

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.

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.

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.

Researchers have developed a new gene therapy that could eventually provide an alternative treatment for Fuchs’ endothelial corneal dystrophy, a genetic eye disease affecting roughly one in 2,000 people globally.

A form of gene therapy protects optic nerve cells and preserves vision in mouse models of glaucoma, according to research supported by NIH’s National Eye Institute. The findings suggest a way forward for developing neuroprotective therapies for glaucoma.

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.

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.

A broadly applicable AAV genome-coupled immunomodulation strategy helps cloak the AAV virus from unwanted immune responses, and offers important insights into ocular inflammation.