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In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels

19 May 2021

Abstract excerpt

Most known pathogenic point mutations in humans are C•G to T•A substitutions, which can be directly repaired by adenine base editors (ABEs). In this study, we investigated the efficacy and safety of ABEs in the livers of mice and cynomolgus macaques for the reduction of blood low-density lipoprotein (LDL) levels. Lipid nanoparticle-based delivery of mRNA encoding an ABE and a single-guide RNA targeting PCSK9, a...

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PMID: 34012094
DOI: 10.1038/s41587-021-00933-4

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