Nature Biotechnology · Jan 12, 2026

Targeted delivery of genome editors in vivo

By Ngo W, Wu JLY, Wasko KM et al.

Genome editing has revolutionized the treatment of genetic diseases, yet the difficulty of tissue-specific delivery currently limits applications of editing technology. In this Review, we discuss preclinical and clinical advances in delivering genome editors with both established and emerging delivery mechanisms. Targeted delivery promises to considerably expand the therapeutic applicability of genome editing, moving closer to the ideal of a precise 'magic bullet' that safely and effectively treats diverse genetic disorders.

doi.org/10.1038/s41587-025-02945-w Open access PubMed 41526513 Interventions