A Phase 1/2 trial included 15 individuals in total, among them three pediatric patients. The patients all suffered from the disease Leber congenital amaurosis caused by a gene that is essential in making proteins crucial to vision.
Gene Editing
The discovery, made in the lab of David Liu, a member of the Broad Core Institute, may one day aid in developing a single gene treatment for illnesses like cystic fibrosis.
MOBEs provide a new tool for disease modelling to researchers.
