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 h...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.展开更多
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).
文摘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.
