Gene editing treats infant's metabolic disorder successfully

A personalized gene editing treatment successfully corrected a life-threatening metabolic disorder in an infant, marking a world-first medical achievement. The therapy used base editing to correct a genetic mutation without cutting DNA. Researchers used base editing to treat an infant with carbamoyl-phosphate synthetase 1 (CPS1) deficiency, a rare genetic disorder. This involved chemically correcting the underlying mutation, offering a precise treatment approach for ultrarare conditions. This innovative treatment, detailed in the New England Journal of Medicine, offers hope for patients with CPS1 deficiency. Further studies are needed to assess long-term effects and expand the treatment to more patients.