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  • RESEARCH SUMMARY

Base editor treats progeria in mice

A single dose of an adenine base editor shows promise in treating the ageing-related disease Hutchinson–Gilford progeria syndrome. It corrected the LMNA mutation underlying the disease in patient-derived cells and improved cardiovascular health and lifespan in mice.
  1. Luke W. Koblan

    1. Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.

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  2. Michael R. Erdos

    1. National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.

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  3. Leslie B. Gordon

    1. Department of Pediatrics, Division of Genetics, Hasbro Children’s Hospital and Warren Alpert Medical School of Brown University, Providence, RI, USA.

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  4. Francis S. Collins

    1. National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.

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  5. Jonathan D. Brown

    1. Vanderbilt University Medical Center, Division of Cardiovascular Medicine, Nashville, TN, USA.

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  6. David R. Liu

    1. Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.

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This is a summary of: Koblan, L. W. et al. In vivo base editing rescues Hutchinson–Gilford progeria syndrome in mice. Nature 589, 608–614 (2021).

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doi: https://doi.org/10.1038/d41586-021-01114-8

References

  1. Eriksson, M. et al. Nature 423, 293–298 (2003).

    PubMed  Article  Google Scholar 

  2. De Sandre-Giovannoli, A. et al. Science 300, 2055 (2003).

    PubMed  Article  Google Scholar 

  3. Gordon, L. B., Brown, W. T. & Collins, F. S. Hutchinson–Gilford Progeria Syndrome (GeneReviews, 2019).

    Google Scholar 

  4. Gordon, L. B. et al. Proc. Natl Acad. Sci. USA 109, 16666–16671 (2012).

    PubMed  Article  Google Scholar 

  5. Gaudelli, N. M. et al. Nature 551, 464–471 (2017).

    PubMed  Article  Google Scholar 

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

    PubMed  Article  Google Scholar 

  7. Grimm, D. & Zolotukhin, S. Mol. Ther. 23, 1819–1831 (2015).

    PubMed  Article  Google Scholar 

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Competing Interests

D.R.L. is a co-founder of Beam Therapeutics, Prime Medicine, Pairwise Plants and Editas Medicine, companies that use genome editing.

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