In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels.
Abstract
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 negative regulator of LDL, induced...
Paper Details
Title
In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels.
DOI
Published Date
May 19, 2021
Journal
Volume
39
Issue
8
Pages
949 - 957
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Notes
History