Targeted delivery of genome editors in vivo

Wayne Ngo; Jamie L Y Wu; Kevin M Wasko; Jennifer A Doudna

doi:10.1038/s41587-025-02945-w

Targeted delivery of genome editors in vivo

Nat Biotechnol. 2026 Jan;44(1):49-59. doi: 10.1038/s41587-025-02945-w. Epub 2026 Jan 12.

Authors

Wayne Ngo^{1

2

3}, Jamie L Y Wu^{1

2

3}, Kevin M Wasko^{1

4}, Jennifer A Doudna^{5

6

7

8

9

10

11

12

13}

Affiliations

¹ Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA, USA.
² Gladstone Institute of Data Science and Biotechnology, San Francisco, CA, USA.
³ California Institute for Quantitative Biosciences, University of California, Berkeley, Berkeley, CA, USA.
⁴ Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA, USA.
⁵ Innovative Genomics Institute, University of California, Berkeley, Berkeley, CA, USA. doudna@berkeley.edu.
⁶ Gladstone Institute of Data Science and Biotechnology, San Francisco, CA, USA. doudna@berkeley.edu.
⁷ California Institute for Quantitative Biosciences, University of California, Berkeley, Berkeley, CA, USA. doudna@berkeley.edu.
⁸ Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA, USA. doudna@berkeley.edu.
⁹ Howard Hughes Medical Institute, University of California, Berkeley, Berkeley, CA, USA. doudna@berkeley.edu.
¹⁰ Molecular Biophysics and Integrated Bioimaging Division, Lawrence Berkeley National Laboratory, Berkeley, CA, USA. doudna@berkeley.edu.
¹¹ Department of Chemistry, University of California, Berkeley, Berkeley, CA, USA. doudna@berkeley.edu.
¹² Li Ka Shing Center for Genomic Engineering, University of California, Berkeley, Berkeley, CA, USA. doudna@berkeley.edu.
¹³ Gladstone-UCSF Institute of Genomic Immunology, San Francisco, CA, USA. doudna@berkeley.edu.

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.

Publication types

Review

MeSH terms

Animals
CRISPR-Cas Systems / genetics
Gene Editing* / methods
Gene Transfer Techniques*
Genetic Therapy* / methods
Humans

Abstract

Publication types

MeSH terms

Grants and funding