Gene Therapy Progress in Leber Congenital Amaurosis

Published 2024-09-12

Researchers from the Perleman School of Medicine and the University of Pennsylvania recently published their findings of a gene therapy trial for Leber Congenital Amaurosis in the journal Lancet. A total of 15 patients with the mutations in the GUCY2D gene participated in this Phase 1/2 clinical trial. All participants had severe vision loss. At the end of the clinical trial, vision improved between 100 fold to 10,000 fold.