“This research demonstrates that CRISPR gene therapy for inherited vision loss is worth continued pursuit in research and clinical trials,” said Pierce, director of the Ocular Genomics Institute and Berman-Gund Laboratory for the Study of Retinal Degenerations at Mass Eye and Ear and Harvard Medical School. “While more research is needed to determine who may benefit most, we consider the early results promising. Hearing from several participants about their excitement at finally being able to see the food on their plates is a significant milestone.”
The clinical trial involved 14 participants, including 12 adults (ages 17 to 63) and two children (ages 10 and 14), all born with a form of Leber Congenital Amaurosis (LCA) caused by mutations in the centrosomal protein 290 (CEP290) gene. They received a single injection of a CRISPR/Cas9 genome editing medicine, EDIT-101, in one eye through a specialized surgical procedure. This trial, which marked the first time a CRISPR-based investigational medicine was administered directly inside the body, primarily assessed safety while also evaluating efficacy.
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