UCSF BENIOFF CHILDREN'S HOSPITAL OAKLAND

CRISPR Clinical Trial at UCSF Targets Sickle Cell Disease at Its Genetic Roots

- UCSF Benioff Children's Hospital Oakland is conducting a clinical trial using CRISPR-Cas9 to correct the genetic mutation causing sickle cell disease. - The trial involves extracting, editing, and reinfusing a patient's blood stem cells, offering a potential cure without needing donor stem cells. - The CRISPR-SCD001 therapy, developed by a UC consortium, aims to establish a healthy blood system free from sickle cell complications. - The trial represents a collaboration between UCSF, UCLA, and the Innovative Genomics Institute, with plans to follow participants for up to 15 years.