CRISPR “One Shot" Cell Therapy for Hemophilia Developed

Scientists at the Salk Institute have combined CRISPR-Cas9 gene editing with stem cell technology to generate a one-time, autologous cell therapy for the genetic blood clotting disorder hemophilia B. In vivo tests showed that gene-edited, stem cell–derived liver cells remained viable and functional in hemophiliac mice for nearly a year, after just a single injection.Headed by Suvasini Ramaswamy, Ph.D., and Inder M. Verma, Ph.D., the Salk Institute team's results offer proof of concept for