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THERAPY

Therapeutic base editing in the adult liver

Gene editing to correct inherited liver disorders has promise for future therapeutic intervention, but lack of effective and safe delivery of the gene-editing machinery to hepatocytes complicates its clinical application. Two studies now report efficient delivery to the liver of non-human primates, providing proof of concept for novel treatment of inherited hypercholesterolaemia.

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Fig. 1: Therapeutic base editing in the liver of macaques upon delivery by lipid nanoparticles.

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Correspondence to Piter J. Bosma.

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Paulusma, C.C., Bosma, P.J. Therapeutic base editing in the adult liver. Nat Rev Gastroenterol Hepatol 18, 597–598 (2021). https://doi.org/10.1038/s41575-021-00491-9

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