UCSF BENIOFF CHILDREN'S HOSPITAL OAKLAND

UCSF Benioff Children's Hospital Oakland is conducting a clinical trial using non-viral CRISPR-Cas9 gene-editing technology to cure sickle cell disease by correcting the genetic mutation in patients' blood stem cells, followed by bone marrow transplant. The trial aims to eliminate the need for a donor and provide a life-long cure. It involves treating up to six adults initially, with potential expansion to adolescents if safe and effective.

A UCSF clinical trial uses CRISPR-Cas9 to edit sickle cell disease-causing mutations in patients' blood stem cells, offering a potential cure without donor stem cells. The trial, a collaboration between UCSF, UCLA, and the Innovative Genomics Institute, aims to reinfuse corrected cells via bone marrow transplant, with initial safety evaluations planned for six adult patients.