IGI Seminar Series – Correction of the Sickle Cell Disease Mutation with CRISPR-Cas9

Sickle cell disease is a hereditary blood disorder in which affected red blood cells contort into a sickle shape. These misshapen cells have shorter lifespans and can block blood flow, leading to symptoms including infection, pain, and fatigue. Currently, the only available cure is an allogeneic bone marrow transplant—a treatment that’s not widely accessible to everyone.

UCLA project scientist Mark DeWitt discusses a combined IGI, UCLA, and UCSF effort in developing a CRISPR-based clinical product to correct sickle cell disease. Listen to Mark as he describes how his team went from a bench genome-editing protocol to an accepted Investigational New Drug (IND).