Gene Therapy Improves Vision In Patients With Leber Congenital Amaurosis, Study Shows

May 07, 2024

CNN (5/6, Howard) reports, “A treatment that used CRISPR was found to be safe and efficacious in improving vision among a small sample of patients with inherited blindness in the Phase 1/2 clinical trial.” Out of 14 participants, “the gene-editing tool was found to be associated with a ‘meaningful improvement’ in vision for most patients around three months later and it was not directly tied to any serious side effects, according to the trial results, published Monday in the New England Journal of Medicine.” The trial “enrolled 12 adults, ages 17 to 63, and two children, ages 9 and 14, with inherited retinal degeneration caused by mutations in the CEP290 gene.”