Advancements in Gene Therapy for Rare Disorders
May 16, 2025
A team of doctors in the U.S. used gene editing to treat a baby with a rare genetic disorder known as carbamoyl phosphate synthetase 1 deficiency. The treatment helped the baby, K.J. Muldoon, begin to develop normally. Scientists are hopeful that this progress will enable the treatment of other rare disorders in the future. The baby's parents had concerns but now feel hopeful.