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.
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.
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.
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.