CRISPR-Cas9 Targeting of <i>PCSK9</i> in Human Hepatocytes In Vivo—Brief Report

Abstract

This work yields important information on the efficacy and safety of CRISPR-Cas9 therapy targeting the human PCSK9 gene in human hepatocytes in vivo, and it establishes humanized mice as a useful platform for the preclinical assessment of applications of somatic in vivo genome editing.