In Vivo Base Editing for Neonatal Inborn Errors of Metabolism: Clinical Progress, N-of-1 Therapy, and the Ethics of Bespoke Genetic Medicine
This review synthesizes the clinical rationale for genetic correction of neonatal IEMs, the mechanistic basis and delivery strategies that make in vivo base editing feasible, and the preclinical evidence that preceded the first human case and examines the ethical dimensions of bespoke 'N-of-1' genetic medicine.