In vivo genome and base editing of a human PCSK9 knock-in hypercholesterolemic mouse model

Published on Dec 1, 2019in BMC Biology6.72
· DOI :10.1186/s12915-018-0624-2
Alba Carreras4
Estimated H-index: 4
(Sahlgrenska University Hospital),
Luna Simona Pane2
Estimated H-index: 2
+ 17 AuthorsMarcello Maresca6
Estimated H-index: 6
Background Plasma concentration of low-density lipoprotein (LDL) cholesterol is a well-established risk factor for cardiovascular disease. Inhibition of proprotein convertase subtilisin/kexin type 9 (PCSK9), which regulates cholesterol homeostasis, has recently emerged as an approach to reduce cholesterol levels. The development of humanized animal models is an important step to validate and study human drug targets, and use of genome and base editing has been proposed as a mean to target disease alleles.
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