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GENOME EDITING

CRISPR base editing lowers cholesterol in monkeys

Nature Biotechnology volume 39, pages 920–921 (2021)Cite this article

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Targeting a gene linked to cardiovascular disease shows therapeutic promise.

CRISPR base editors are advancing rapidly toward the clinic with the publication of two studies demonstrating successful reduction of blood cholesterol in monkeys. The papers, by Rothgangl et al.1 in Nature Biotechnology and Musunuru et al.2 in Nature, show that transient expression of base editors to knock out a gene associated with cardiovascular disease is effective and safe in non-human primates. Delivery of the base editors to the liver in lipid nanoparticles led to efficient knockout of the target gene—proprotein convertase subtilisin/kexin type 9 (PCSK9)—and significant reductions of blood cholesterol, with no substantial off-target editing or serious immune responses. These impressive results suggest that the first cardiovascular gene-editing therapy could rapidly become a reality once the approach is fine-tuned for human testing.

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Fig. 1: Delivery of CRISPR base editing components in lipid nanoparticles for precise genome editing.

References

  1. Rothgangl, T. et al. Nat. Biotechnol. https://doi.org/10.1038/s41587-021-00933-4 (2021).

    Article  PubMed  PubMed Central  Google Scholar 

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

    Article  CAS  PubMed  Google Scholar 

  3. Zhang, D. W. et al. J. Biol. Chem. 282, 18602–18612 (2007).

    Article  CAS  PubMed  Google Scholar 

  4. Abifadel, M. et al. Nat. Genet. 34, 154–156 (2003).

    Article  CAS  PubMed  Google Scholar 

  5. Cohen, J. et al. Nat. Genet. 37, 161–165 (2005).

    Article  CAS  PubMed  Google Scholar 

  6. First in vivo human genome editing trial. Nat. Biotechnol. 36, 5 (2018).

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

    Article  CAS  PubMed  Google Scholar 

  8. Jin, S. et al. Science 364, 292–295 (2019).

    CAS  PubMed  Google Scholar 

  9. Zuo, E. et al. Science 364, 289–292 (2019).

    CAS  PubMed  PubMed Central  Google Scholar 

  10. Chadwick, A. C., Wang, X. & Musunuru, K. Arterioscler. Thromb. Vasc. Biol. 37, 1741–1747 (2017).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  11. Gaudelli, N. M. et al. Nat. Biotechnol. 38, 892–900 (2020).

    Article  CAS  PubMed  Google Scholar 

  12. Hanlon, K. S. et al. Nat. Commun. 10, 4439 (2019).

    Article  PubMed  PubMed Central  Google Scholar 

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Authors and Affiliations

  1. Hubrecht Institute, KNAW and University Medical Center Utrecht, Utrecht, the Netherlands

    Sebastiaan Johannes van Kampen & Eva van Rooij

  2. Department of Cardiology, University Medical Center Utrecht, Utrecht, the Netherlands

    Eva van Rooij

Authors
  1. Sebastiaan Johannes van Kampen
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  2. Eva van Rooij
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Correspondence to Eva van Rooij.

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The authors declare no competing interests.

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van Kampen, S.J., van Rooij, E. CRISPR base editing lowers cholesterol in monkeys. Nat Biotechnol 39, 920–921 (2021). https://doi.org/10.1038/s41587-021-00975-8

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