Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • News & Views
  • Published:

Genetic diversity

Accounting for diversity in the design of CRISPR-based therapeutic genome editing

Nature Genetics volume 55, pages 6–7 (2023)Cite this article

Subjects

CRISPR cell and gene therapy have been designed largely with respect to a single reference human genome. A new study reveals how human genetic diversity could lead to off-target effects and presents a new tool to identify these risks.

This is a preview of subscription content, access via your institution

Access options

Buy this article

  • Purchase on SpringerLink
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

Fig. 1: Assaying human genetic diversity can advance the design of human therapeutics involving genome editing.

References

  1. Graham, F. Nature https://doi.org/10.1038/d41586-020-01928-y (2020).

  2. Science News Staff. Science https://doi.org/10.1126/science.adf7363 (2022).

  3. Cancellieri, S. et al. Nat. Genet. https://doi.org/10.1038/s41588-022-01257-y (2022).

  4. Sinha, D. et al. Am. J. Hum. Genet. 107, 278–292 (2020).

    Article  CAS  Google Scholar 

  5. Keough, K. C. et al. Genome Biol. 20, 167 (2019).

    Article  Google Scholar 

  6. Saha, K. et al. Nature 592, 195–204 (2021).

    Article  CAS  Google Scholar 

  7. Misek, S. A. et al. Preprint at bioRxiv https://doi.org/10.1101/2022.11.18.517155 (2022).

  8. Lessard, S. et al. Proc. Natl Acad. Sci. USA 114, E11257–E11266 (2017).

    Article  CAS  Google Scholar 

  9. Scott, D. A. & Zhang, F. Nat. Med. 23, 1095–1101 (2017).

    Article  CAS  Google Scholar 

  10. Leibowitz, M. L. et al. Nat. Genet. 53, 895–905 (2021).

    Article  CAS  Google Scholar 

  11. Maeder, M. L. et al. Nat. Med. 25, 229–233 (2019).

    Article  CAS  Google Scholar 

  12. Musunuru, K. et al. Nature 593, 429–434 (2021).

    Article  CAS  Google Scholar 

  13. Foy, S. P. et al. Nature https://doi.org/10.1038/s41586-022-05531-1 (2022).

  14. Center for Biologics Evaluation and Research. Human Gene Therapy Products Incorporating Human Genome Editing (U.S. Food and Drug Administration, 2022).

Download references

Acknowledgements

K.S. has received funding from the US National Science Foundation, grant nos. EEC-1648035 and AWD-101645-G3/RJ375-G3, and the US National Institutes for Health, grant nos. R35GM119644, U01EY032333 and UH3NS111688.

Author information

Authors and Affiliations

  1. Department of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI, USA

    Krishanu Saha

  2. McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI, USA

    Krishanu Saha

  3. Wisconsin Institute for Discovery, University of Wisconsin-Madison, Madison, WI, USA

    Krishanu Saha

  4. Department of Medical History & Bioethics, University of Wisconsin-Madison, Madison, WI, USA

    Krishanu Saha

Authors
  1. Krishanu Saha
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Krishanu Saha.

Ethics declarations

Competing interests

K.S. receives sponsored research support from Spotlight Therapeutics and Synthego and is an advisor to Notch Therapeutics and Andson Biotech.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Saha, K. Accounting for diversity in the design of CRISPR-based therapeutic genome editing. Nat Genet 55, 6–7 (2023). https://doi.org/10.1038/s41588-022-01272-z

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/s41588-022-01272-z

Search

Advanced search

Quick links

Nature Briefing: Translational Research

Sign up for the Nature Briefing: Translational Research newsletter — top stories in biotechnology, drug discovery and pharma.

Get what matters in translational research, free to your inbox weekly. Sign up for Nature Briefing: Translational Research