The Center will use CRISPR-based editing technology to advance cures for severe pediatric genetic diseases and will bridge CRISPR cure design and testing at the University of California, Berkeley (UC Berkeley) with clinical treatment at the University of California, San Francisco (UCSF) - both partners in the MRC CoRE.

Selected news coverage: Endpoints news, Time, Ground Truths podcast

MRC CoRE members at the University of Berkeley were central to delivering this therapy, working closely with a wider team led by the Children's Hospital of Philadelphia.

YouTube video by Children's Hospital of Philadelphia.

Original scientific publication

A team of physicians and scientists including five researchers from the Innovative Genomics Institute (IGI) at the University of California, Berkeley, achieved a historic milestone for the field: a personalized in vivo CRISPR therapy for an infant was developed and delivered to the patient in just six months. The patient, KJ, was born with a rare, severe genetic disease that affects just 1 in 1.3 million newborns. This landmark case paves the way for a future with on-demand gene-editing therapies for individuals with rare, until-now untreatable genetic diseases.

Selected news coverage: The Economist, New York Times, Time, Nature, UC Berkeley IGI, Children's Hospital of Philadelphia

James Coney, father of Charlie, wrote a long piece in The Times, talking about his family's experience of receiving a genetic diagnosis.