World First: Baby Treated with Personalized CRISPR Gene Therapy is Now Thriving

Good News Network reports on a groundbreaking medical achievement: a baby girl named KJ became the first person ever to receive a personalized CRISPR-based gene therapy. Born with carbamoyl phosphate synthetase 1 deficiency (CPS1), a rare metabolic disorder that prevents the body from processing nitrogen, KJ faced a grim prognosis.

Doctors at Children's Hospital of Philadelphia developed a custom treatment in record time. The therapy uses CRISPR technology to correct the genetic defect in her liver cells. Months after treatment, KJ is thriving — eating normally, growing, and hitting developmental milestones.

“Born with carbamoyl phosphate synthetase 1 deficiency (CPS1), a rare metabolic disorder that prevents the body from processing nitrogen, KJ faced a grim prognosis.”