Everything has to go right for a life-saving transplant. Too often, the system makes it impossible. Sixty-six-year-old Brian ...

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

For the first time, doctors have created a customized treatment using the revolutionary gene-editing technique known as ...

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 ...

G ene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments has ...

A new gene editor takes advantage of CRISPR-associated proteins to insert whole genes into the genome, scientists report.

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