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 the potential use of autologous cell therapy in the treatment of hemophilia B and potentially other liver disorders that are similarly caused by defects in a single gene. “The appeal of a cell-based approach is that you minimize the number of treatments that a patient needs,” says Dr. Ramaswamy, a former Salk research associate in laboratory of Dr. Verma, and first author of the team’s paper published in Cell Reports.  “Rather than constant …