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.

GENOME EDITING

‘Hit and run’ therapy averts macular degeneration

Subretinal delivery of lentiviruses bearing Cas9 mRNA and a guide RNA targeting the Vegfa gene reduces the development of choroidal neovascularization in a mouse model of wet age-related macular degeneration.

Your institute does not have access to this article

Access options

Buy article

Get time limited or full article access on ReadCube.

$32.00

All prices are NET prices.

Fig. 1: Lentiviral delivery of Cas9 mRNA and a Vegfa-targeting gRNA to retinal epithelium largely prevents the development of wet age-related macular degeneration in mice.

References

  1. Prel, A. et al. Mol. Ther. Methods Clin. Dev. 2, 15039 (2015).

    Article  Google Scholar 

  2. Ling, S. et al. Nat. Biomed. Eng. https://doi.org/10.1038/s41551-020-00656-y (2021).

  3. Michel, G., Yu, Y., Chang, T. & Yee, J. K. Mol. Ther. 18, 1814–1821 (2010).

    CAS  Article  Google Scholar 

  4. Cai, Y. et al. Nucleic Acids Res. 42, e28 (2014).

    CAS  Article  Google Scholar 

  5. He, C. et al. Gene Ther. 21, 759–766 (2014).

    CAS  Article  Google Scholar 

  6. Cai, Y., Bak, R. O. & Mikkelsen, J. G. eLife 3, e01911 (2014).

    Article  Google Scholar 

  7. Lyu, P., Wang, L. & Lu, B. Life (Basel) 10, 366 (2020).

    Google Scholar 

  8. Yin, D. et al. Nat. Biotechnol. https://doi.org/10.1038/s41587-020-00781-8 (2021).

  9. Arthur, L. O. et al. Science 258, 1935–1938 (1992).

    CAS  Article  Google Scholar 

  10. Rulli, S. J.Jr et al. J. Virol. 81, 6623–6631 (2007).

    CAS  Article  Google Scholar 

  11. Nguyen, G. N. et al. Nat. Biotechnol. 39, 47–55 (2021).

    CAS  Article  Google Scholar 

  12. Anzalone, A. V., Koblan, L. W. & Liu, D. R. Nat. Biotechnol. 38, 824–844 (2020).

    CAS  Article  Google Scholar 

  13. Wesselhoeft, R. A., Kowalski, P. S. & Anderson, D. G. Nat. Commun. 9, 2629 (2018).

    Article  Google Scholar 

  14. Li, A. et al. Mol. Ther. 28, 1432–1441 (2020).

    CAS  Article  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Dirk Grimm.

Ethics declarations

Competing interests

D.G. is a co-founder and shareholder of AaviGen GmbH. F.B. and D.G. have filed patent applications related to CRISPR technologies and gene delivery via adeno-associated viruses.

Rights and permissions

Reprints and Permissions

About this article

Verify currency and authenticity via CrossMark

Cite this article

Bubeck, F., Grimm, D. ‘Hit and run’ therapy averts macular degeneration. Nat Biomed Eng 5, 132–133 (2021). https://doi.org/10.1038/s41551-021-00690-4

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/s41551-021-00690-4

Search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing