UC Consortium Launches First Clinical Trial Using CRISPR to Correct Gene Defect That Causes Sickle Cell Disease Dr. Mark Walters of UCSF talks about how those who suffer from sickle cell disease, could be helped by a new CRISPR gene therapy developed by scientists and physicians at UC Berkeley, UCSF and UCLA. The trial aims to directly correct the sickle mutation in blood stem cells that causes them to create deformed red blood cells, leading to the debilitating and painful disease, according to Mark Walters, MD, a professor of pediatrics at UCSF and principal investigator of the clinical trial and gene editing project. It will be the first time clinical researchers attempt to correct the faulty beta-globin gene in a patient's own cells with non-virally delivered CRISPR gene correction tools. Series: "UC Berkeley News" [Health and Medicine] [Show ID: 37368]