Targeted delivery of genome editors in vivo.
review · Level V
Where this comes from
- Record sourced from PubMed, PMID 41526513.
- Also identified by DOI 10.1038/s41587-025-02945-w and PMC identifier 12875382.
- Licence recorded as CC BY.
- The licence permits redistribution, so the abstract is shown in full and the full text is available from the publisher.
Abstract
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.
Medical subject headings
- Gene Editing
- Genetic Therapy
- Gene Transfer Techniques
- Drug Delivery Systems