Infant born with deadly disease now thriving thanks to customized CRISPR treatment six months after birth
A newborn boy, KJ, was diagnosed at birth with a rare and potentially fatal genetic disease. He received a novel, on-demand CRISPR therapy in record time.
Researchers at the University of California, Berkeley’s Innovative Genomics Institute (IGI) worked with the boy’s physicians at the Children’s Hospital of Philadelphia (CHOP) to test the safety and efficacy of a CRISPR base-editing therapy that hospital doctors had developed.
“A remarkable multi-institution team — CHOP, Penn, IGI/UC Berkeley, IDT, Aldevron, Jackson Labs — came together to show that one can create a CRISPR on-demand therapy in six months. It can be done even faster. Now, we have to show that we can scale this from CRISPR for one to CRISPR for all.”
https://news.berkeley.edu/2025/05/15/infant-born-with-deadly-disease-now-thriving-thanks-to-customized-crispr-treatment-six-months-after-birth/