Article
CRISPR-Cas9 Targeting of PCSK9 in Human Hepatocytes In Vivo-Brief Report.
Arteriosclerosis, thrombosis, and vascular biology - 1 May 2016
Wang Xiao, Raghavan Avanthi, Chen Tao, Qiao Lyon, Zhang Yongxian, Ding Qiurong, Musunuru Kiran
Abstract excerpt
OBJECTIVE: 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...
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