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Article

In Vivo Base Editing of PCSK9 (Proprotein Convertase Subtilisin/Kexin Type 9) as a Therapeutic Alternative to Genome Editing.

Arteriosclerosis, thrombosis, and vascular biology - 1 Sept 2017

Chadwick Alexandra C, Wang Xiao, Musunuru Kiran

Abstract excerpt

OBJECTIVE: High-efficiency genome editing to disrupt therapeutic target genes, such as PCSK9 (proprotein convertase subtilisin/kexin type 9), has been demonstrated in preclinical animal models, but there are safety concerns because of the unpredictable nature of cellular repair of double-strand breaks, as well as off-target mutagenesis. Moreover, precise knock-in of specific nucleotide changes-whether to...

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Source

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Identifiers

PMID: 28751571
DOI: 10.1161/ATVBAHA.117.309881
PMCID: PMC5570639

Genes

PCSK9

Provenance

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