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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).

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