In vivo PCSK9 gene editing using an all-in-one self-cleavage AAV-CRISPR system
Qian Li, Jing Su, Yi Liu, Xiu Jin, Xiaomei Zhong, Li Mo, Qingnan Wang, Hongxin Deng, Yang Yang
Abstract
. Moreover, the on-target editing efficacy was not compromised and resulted in a stable reduction in circulating PCSK9 and serum cholesterol. The inclusion of this self-cleavage system in gene-editing approaches could increase the safety profile of AAV-delivered genome-editing nucleases and thereby promote its clinical transformation.
Topics & Concepts
CRISPRGenome editingPCSK9In vivoGeneCleavage (geology)BiologyComputational biologyGeneticsLDL receptorCholesterolBiochemistryFracture (geology)LipoproteinPaleontologyCRISPR and Genetic EngineeringViral Infections and Immunology ResearchViral Infectious Diseases and Gene Expression in Insects