UCSF researchers hope to cure sickle cell disease through CRISPR gene editing

With a $17 million grant, UCSF Benioff Children’s Hospital in Oakland will launch a four-year trial evaluating CRISPR/Cas9 gene editing as a potential cure for sickle cell disease. The trial is the first to evaluate the use of non-viral CRISPR gene editing tools to correct a harmful mutation in the beta-globin gene in a patient’s own cells. The trial is part of a UC Research consortium led by UCSF in collaboration with UCLA and UC Berkeley. It will initially enroll up to six adults with sickle cell disease at sites in Oakland and Los Angeles and, after meeting safety and efficacy criteria, will expand to include three adolescents aged 12 to 17 years. Seven patients will be treated in Oakland and two in Los Angeles.  During the trial, researchers will extract stem cells from a patient’s blood and send them to UCLA’s gene manufacturing lab for processing. The trial will use CRISPR technology from the Innovative Genomics Institute (IGI), a UC Berkley-UCSF collaboration founded by Doudna. They will use electrical pulses to create temporary pores in the cell membranes. This will facilitate the entry of the CRISPR/Cas9 platform into the cells and ultimately into the nucleus, where it corrects the cell mutation before being returned to the patient.  

UCSF researchers hope to cure sickle cell disease through CRISPR gene editing | Genetic Engineering in the Press by GEG | Scoop.it

Share :

Tags :