Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors

Andrew V Anzalone^#^{1

2

3}, Luke W Koblan^#^{1

2

3}, David R Liu^{4

5

6}

Affiliations

¹ Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
² Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.
³ Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA.
⁴ Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA. drliu@fas.harvard.edu.
⁵ Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA. drliu@fas.harvard.edu.
⁶ Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA. drliu@fas.harvard.edu.

^# Contributed equally.

PMID: 32572269
DOI: 10.1038/s41587-020-0561-9

Review

Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors

Andrew V Anzalone et al. Nat Biotechnol. 2020 Jul.

. 2020 Jul;38(7):824-844.

doi: 10.1038/s41587-020-0561-9. Epub 2020 Jun 22.

Authors

Andrew V Anzalone^#^{1

2

3}, Luke W Koblan^#^{1

2

3}, David R Liu^{4

5

6}

Affiliations

¹ Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
² Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.
³ Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA.
⁴ Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA. drliu@fas.harvard.edu.
⁵ Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA. drliu@fas.harvard.edu.
⁶ Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA. drliu@fas.harvard.edu.

^# Contributed equally.

PMID: 32572269
DOI: 10.1038/s41587-020-0561-9

Abstract

The development of new CRISPR-Cas genome editing tools continues to drive major advances in the life sciences. Four classes of CRISPR-Cas-derived genome editing agents-nucleases, base editors, transposases/recombinases and prime editors-are currently available for modifying genomes in experimental systems. Some of these agents have also moved rapidly into the clinic. Each tool comes with its own capabilities and limitations, and major efforts have broadened their editing capabilities, expanded their targeting scope and improved editing specificity. We analyze key considerations when choosing genome editing agents and identify opportunities for future improvements and applications in basic research and therapeutics.

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References

1. Jinek, M. et al. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 337, 816–821 (2012). - PubMed - PMC
1. Gasiunas, G., Barrangou, R., Horvath, P. & Siksnys, V. Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria. Proc. Natl Acad. Sci. USA 109, E2579–E2586 (2012). - PubMed - PMC
1. Cong, L. et al. Multiplex genome engineering using CRISPR/Cas systems. Science 339, 819–823 (2013). - PubMed - PMC
1. Mali, P. et al. RNA-guided human genome engineering via Cas9. Science 339, 823–826 (2013). - PubMed - PMC
1. Hwang, W. Y. et al. Efficient genome editing in zebrafish using a CRISPR-Cas system. Nat. Biotechnol. 31, 227–229 (2013). - PubMed - PMC

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Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors

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Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors

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