Gene therapy improves vision in Leber congenital amaurosis patients
May 10, 2024
An OHSU study finds about 79% of clinical trial participants with leber congenital amaurosis experienced measurable improvement after receiving experimental, CRISPR-based gene editing: “This [is] a blinding disease with no treatments ... an enormous burden on patients [due] to their vision loss,” says Mark Pennesi, M.D., Ph.D.