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Article

Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9

21 Feb 2021

Abstract excerpt

Gene disruption via programmable, sequence-specific nucleases represents a promising gene therapy strategy in which the reduction of specific protein levels provides a therapeutic benefit. Proprotein convertase subtilisin/kexin type 9 (PCSK9), an antagonist of the low-density lipoprotein (LDL) receptor, is a suitable target for nuclease-mediated gene disruption as an approach to treat hypercholesterolemia. We...

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PMID: 33609733
DOI: 10.1016/j.ymthe.2021.02.020

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