Success in Sight for Gene Editing

The development of efficient new gene-editing techniques has improved the prospect of curative genetic medicines. In 2012, Jinek and colleagues described an adaptive immune system in single-cell organisms, in which clustered regularly interspaced short palindromic repeat (CRISPR) sequences target foreign DNA for cleavage by CRISPR-associated (Cas) proteins, highlighting the potential of this system for gene editing.1 CRISPR-Cas (also referred to as Cas9–guide RNA [gRNA]) technology has since been widely adopted as a versatile laboratory tool. In 2023, the European Medicines Agency and the Food and Drug Administration approved the CRISPR-based therapy exagamglogene autotemcel (Casgevy), which is administered to harvested blood . . .