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Lipid nanoparticles incorporating a GalNAc ligand enable in vivo liver <i>ANGPTL3</i> editing in wild-type and somatic <i>LDLR</i> knockout non-human primates

2021-11-08

Abstract excerpt

Standard lipid nanoparticles (LNPs) deliver gene editing cargoes to hepatocytes through receptor-mediated uptake via the low-density lipoprotein receptor (LDLR). Homozygous familial hypercholesterolemia (HoFH) is a morbid genetic disease characterized by complete or near-complete LDLR deficiency, markedly elevated blood low-density lipoprotein cholesterol (LDL-C) levels, and premature atherosclerotic cardiovascula...

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Literature Corpus work
a3449cf0-fa32-55c4-bc8b-6c3b12e65659
DOI
10.1101/2021.11.08.467731
Open publication

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Lipid nanoparticles incorporating a GalNAc ligand enable in vivo liver <i>ANGPTL3</i> editing in wild-type and somatic <i>LDLR</i> knockout non-human primatesDOI 10.1101/2021.11.08.467731
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