Individualized and Platform-Designated Gene-Editing Studies in the Registered Clinical Record: A Case-by-Case Census Against 148 Eligible Studies
IntroductionIn February 2025, a single infant with carbamoyl phosphate synthetase 1 deficiency received a customized base-editing therapy, and individualized gene editing has since been discussed as an emerging category of care.No one has yet counted whether this category exists in the registered study record. MethodsW...