In a historic first for genetic medicine, doctors and scientists at Children’s Hospital of Philadelphia (CHOP) and Penn Medicine have successfully used a customized CRISPR-based gene editing therapy to treat a baby with a rare, life-threatening metabolic condition. The patient, known as KJ, was born with carbamoyl phosphate synthetase 1 (CPS1) deficiency, a disorder that disrupts the body’s ability to process nitrogen, causing toxic ammonia buildup in the blood.
This is the first time in the world that a CRISPR therapy has been specifically tailored and administered to a single patient, marking a revolutionary advancement in personalized medicine.
Continue reading… “A Genetic Breakthrough: First-Ever Personalized CRISPR Therapy Saves Infant with Rare Metabolic Disorder”