UCSF, Berkeley, UCLA to Launch Sickle Cell Trial Using CRISPR

8/20/2021; 4 minutes

UC Consortium launches first clinical trial using CRISPR to correct the gene defect that causes Sickle Cell Disease. Dr. Mark Walters of UCSF talks about 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. 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. (#37368)

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