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UC group launches clinical trial using CRISPR to correct sickle cell disease gene defect April 1, 2021UCLA Scientists at UCLA, UC San Francisco and UC Berkeley have received U.S. Food and Drug Administration approval to jointly launch an early phase, first-in-human clinical trial of a CRISPR gene correction therapy in patients with sickle cell disease using the patients’ own blood-forming stem cells. The trial will combine CRISPR technology developed at the Innovative Genomics Institute a UC Berkeley–UCSF initiative founded by Berkeley’s Nobel Prize–winning scientist Jennifer Doudna with UCLA’s expertise in genetic analysis and cell manufacturing, and the decades-long expertise at UCSF Benioff Children’s Hospital Oakland in cord blood and marrow transplantation and in gene therapy for sickle cell disease. The four-year study will include six adults and three adolescents with severe sickle cell disease. It is planned to begin this summer in Oakland and Los Angeles.

United statesDonald kohnMark waltersFyodor urnovLos angelesJennifer doudnaCalifornia institute for regenerative medicineBroad stem cell research centerDavid geffen school of medicineDrug administrationCure sickle cell initiativeMarrow transplant programInnovative genomics instituteDoris duke charitable foundationNational heartNobel prize