A baby born with carbamoyl-phosphate synthetase 1 (CPS1) deficiency – an ultra-rare metabolic condition that prevents the body from clearing toxic ammonia, which is often fatal in infancy – has become the first person in the world to be successfully treated with a bespoke genome-editing therapy.
The therapy was developed at the Children's Hospital of Philadelphia.
After multiple infusions of the treatment, beginning at around six months of age, the baby has since grown, gained weight and shown signs of normal development previously unlikely for patients with the disorder.
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