The story worth to track – Infant treated with personalized CRISPR gene editing therapy for CPS1 deficiency.

2024 – “Infant KJ is diagnosed with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, a rare and life-threatening metabolic disorder, just days after his birth.”
2025 – “At approximately six months of age, KJ receives the first infusion of his customized CRISPR base-editing…