UC group launches clinical trial using CRISPR to correct sickle cell disease gene defect

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 an d 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.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 Dr. Mark Walters, 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 tocorrect the faulty beta-globin gene in a patient ’s own cells with non-virally delivered CRISPR gene correction tools. Previously, researchers have used more costly and indirect approaches such as reactivating fetal hemoglobin or us...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news