Neat News 3155 (added 2024-09-09)
Gene therapy for vision in Leber congential amaurosis
© Rhcastilhos [CC BY-SA 3.0]
About 100,000 people suffer from Leber congenital amaurosis due to a mutation in the so-called GUCY2D gene (LCA1). This results in a significant amount of vision loss or even blindness. Now in a small trial of 15 patients a gene therapy has been attempted, their vision was 100 times better after the treatment, two of nine who received the highest dose even had a 10,000 fold improvement.
related: #eye #sight #gene_therapy
