A subretinal gene therapy was well tolerated 1 year after treatment, with no drug-related serious adverse events, in a small group of patients with Leber congenital amaurosis 1 (LCA1), a rare ...

Leber Congenital Amaurosis (LCA) is an inherited retinal disease leading to severe vision impairment from early infancy, affecting 2-3 out of every 100,000 newborns. LCA is caused by variants in ...

Local restaurants, like Applebee’s, are participating in a fundraiser Aug. 5-9 for Leber Congenital Amaurosis research. Leber Congenital Amaurosis, otherwise known as LCA, is a rare genetic eye ...

Please provide your email address to receive an email when new articles are posted on . No serious treatment-emergent adverse events were seen at 12 months. Patients who received a high dose of ...

An experimental gene therapy restored the night vision of two people with leber congenital amaurosis. When you purchase through links on our site, we may earn an affiliate commission. Here’s how it ...

One of the greatest gene therapy success stories to date (which occurred in 2017) was the FDA approval of Luxturna—an AAV-based gene therapy to treat inherited retinal disease. The gene therapy treats ...

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 ...

Gene therapy may restore vision to children and adults robbed of their sight by a rare inherited condition called Leber congenital amaurosis, researchers report. The illness is caused by mutations in ...

After the treatment, one patient saw her first star. Another saw snowflakes for the first time. Other patients were newly able to navigate outside of the home or to read the labels on their child’s ...

Sight loss in certain inherited eye diseases may be caused by gut bacteria, and is potentially treatable by antimicrobials, finds a new study in mice co-led by a UCL and Moorfields researcher. The ...