On May 15, 2025, a research team from the Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania published a landmark study in the New England Journal of Medicine(NEJM): the first successful application of a bespoke gene-editing therapy, developed for a single patient, to treat an infant with a rare and fatal genetic disorder—carbamoyl phosphate synthetase 1 (CPS1) deficiency.

Alzheimer’s disease is a progressive neurodegenerative disorder characterized by a gradual decline in cognitive function, including memory loss, impaired thinking, and behavioral changes. The disease is strongly associated with aging and is most commonly observed in individuals over the age of 65.