
ELSI Friday Forum | Feb. 13 | 12p ET
Just CRISPR: Is interventional genetics for rare diseases scalable? In 2025, KJ Muldoon was the first patient to be treated for a life-threatening genetic disorder with gene editing therapy. KJ received a diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency (CPS1) as a neonate, and received treatment when he was 7 months old, under Expanded Access …


