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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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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DOI: https://doi.org/10.1038/s41575-021-00491-9
