Viral and nonviral nanocarriers for in vivo CRISPR-based gene editing  

作　　者：Zhongyuan Guo Audrey T.Zhu Ronnie H.Fang Liangfang Zhang 

机构地区：[1]Aiiso Yufeng Li Family Department of Chemical and Nano Engineering,University of California San Diego,La Jolla,CA 92093,USA [2]Division of Host-Microbe Systems and Therapeutics,Department of Pediatrics,University of California San Diego,La Jolla,CA 92093,USA

出　　处：《Nano Research》2024年第10期8904-8925,共22页纳米研究（英文版）

基　　金：supported by the Defense Threat Reduction Agency Joint Science and Technology Office for Chemical and Biological Defense(No.HDTRA1-21-1-0010);the National Institutes of Health(Nos.R21AI159492,and R21AI175904).

摘　　要：The continued development of clustered regularly interspaced short palindromic repeats(CRISPR)technology has the potential to greatly impact clinical medicine,particularly for disease diagnosis and treatment.Despite high demand for the in vivo delivery of CRISPR-based therapies,significant challenges persist.These include rapid degradation by enzymes,inefficient disease site targeting,and the risk of undesired off-target outcomes.Nanoparticulate platforms,with their tailorable properties,have been engineered to efficiently package CRISPR payloads in various formats,including as plasmid DNA,mRNA,and ribonucleoprotein complexes,for in vivo delivery.Among them,recombinant adeno-associated viruses,virus-like particles,and lipid nanoparticles have displayed exceptional promise.This review will discuss the development of these and other nanocarriers for in vivo CRISPR-based genome editing.

关 键 词：clustered regularly interspaced short palindromic repeats(CRISPR) in vivo gene editing NANOCARRIERS recombinant adeno-associated viruses(rAAVs) virus-like particle(VLP) lipid nanoparticle(LNP) 

分 类 号：Q78[生物学—分子生物学]