
CRISPR Gene Editing Breakthrough: Personalized Cure for Rare CPS1 Liver Disorder in Infant
Introduction In a landmark achievement in gene therapy, doctors at the Children’s Hospital of Philadelphia (CHOP) and Penn Medicine have successfully used a personalized CRISPR-based gene-editing therapy to treat a nine-month-old infant, KJ Muldoon, diagnosed with a rare and life-threatening liver disorder known as carbamoyl phosphate synthetase 1 (CPS1) deficiency. This pioneering treatment marks the…