Baby Is Healed With World’s First Personalized Gene-Editing Treatment

May 15, 2025

3D rendering of a DNA molecule

(New York Times) – The technique used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other uncommon genetic diseases.

The baby, now 9 ½ months old, became the first patient of any age to have a custom gene-editing treatment, according to his doctors. He received an infusion made just for him and designed to fix his precise mutation.

The investigators who led the effort to save KJ are presenting their work on Thursday at the annual meeting of the American Society of Cell & Gene Therapy, and are also publishing it in the New England Journal of Medicine. (Read More)