KJ received the bespoke CRISPR treatment 6.5 months after birth, receiving three infusions of the therapy as of April 2025.

Just six months after a newborn at Children's Hospital of Philadelphia was diagnosed with a rare and life-threatening metabolic disorder, doctors were able to develop a personalized treatment ...

A baby born with a rare and dangerous genetic disease is thriving after getting an experimental gene editing treatment made ...

Family overjoyed correction to DNA defect that causes metabolic disorder has given little KJ a new lease of life ...

In a study published May 15 in the New England Journal of Medicine and presented at the American Society of Gene and Cell ...