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‘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.

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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.


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

    Article  Google Scholar 

  2. Ling, S. et al. Nat. Biomed. Eng. (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. (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 

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Correspondence to Dirk Grimm.

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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.

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Bubeck, F., Grimm, D. ‘Hit and run’ therapy averts macular degeneration. Nat Biomed Eng 5, 132–133 (2021).

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