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UC Consortium Launches First Clinical Trial Using CRISPR to Correct Gene Defect That Causes Sickle Cell Disease

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Illustration of sickle cells moving through a vein.

Image: Illustration of sickle cells moving through a vein – UCSF. Article by Lorna Fernandes. UCSF – March 30, 2021.

Project Uses Nobel-Prize-Winning Technology to Directly Correct Sickle Mutation in Blood Stem Cells, Addressing Underlying Cause of Debilitating Disorder

Scientists at UC San Francisco, UC Berkeley and UCLA 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 patient’s own blood-forming stem cells.

The trial will combine CRISPR technology developed at Innovative Genomics Institute (IGI) — a joint UC Berkeley-UCSF initiative founded by Berkeley’s Nobel Prize-winning scientist Jennifer Doudna, PhD — 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 4-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. […]

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