CRISPR-Cas9 Targeting of PCSK9 in Human Hepatocytes In Vivo—Brief Report | Zendy

Xiao Wang | Zendy; Avanthi Raghavan | Zendy; Tao Chen | Zendy; Lyon Qiao | Zendy; Yongxian Zhang | Zendy; Qiurong Ding | Zendy; Kiran Musunuru | Zendy

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CRISPR-Cas9 Targeting of PCSK9 in Human Hepatocytes In Vivo—Brief Report

Author(s) -

Xiao Wang,

Avanthi Raghavan,

Tao Chen,

Lyon Qiao,

Yongxian Zhang,

Qiurong Ding,

Kiran Musunuru

Publication year - 2016

Publication title -

arteriosclerosis thrombosis and vascular biology

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 3.007

H-Index - 270

eISSN - 1524-4636

pISSN - 1079-5642

DOI - 10.1161/atvbaha.116.307227

Subject(s) - crispr , genome editing , biology , mutagenesis , pcsk9 , cas9 , human genome , kexin , proprotein convertase , gene targeting , gene , computational biology , somatic cell , genetic enhancement , genome , genetics , mutation , ldl receptor , lipoprotein , biochemistry , cholesterol

Although early proof-of-concept studies of somatic in vivo genome editing of the mouse ortholog of proprotein convertase subtilisin/kexin type 9 (Pcsk9) in mice have established its therapeutic potential for the prevention of cardiovascular disease, the unique nature of genome-editing technology-permanent alteration of genomic DNA sequences-mandates that it be tested in vivo against human genes in normal human cells with human genomes to give reliable preclinical insights into the efficacy (on-target mutagenesis) and safety (lack of off-target mutagenesis) of genome-editing therapy before it can be used in patients.

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