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In vivo base editing rescues progeria in mice

  1. Megan Cully
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Base editing enables single-base corrections in genomes and could therefore be useful in correcting pathogenic gene variants, approximately half of which are point mutations. Hutchinson–Gilford progeria syndrome (HGPS) is a fatal genetic disorder characterized by rapid aging, cardiovascular disease and early death. Writing in Nature, Luke Koblan and colleagues used an adenine base editor (ABE) to correct the HGPS-causing mutation. In fibroblasts derived from patients with HGPS, treatment with this ABE substantially reduced the expression of the mutant protein. In mouse models, the ABE efficiently edited this locus, prevented the abnormal cardiovascular histology associated with this disease and greatly extended lifespan.

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Nature Reviews Drug Discovery 20, 98 (2021)

doi: https://doi.org/10.1038/d41573-021-00013-x

References

  1. Koblan, L. W. et al. In vivo base editing rescues Hutchinson–Gilford progeria syndrome in mice. Nature https://doi.org/10.1038/s41586-020-03086-7 (2021)

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