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

被引:0
|
作者
Guo, Zhongyuan [1 ]
Zhu, Audrey T. [1 ]
Fang, Ronnie H. [1 ,2 ]
Zhang, Liangfang [1 ]
机构
[1] Univ Calif San Diego, Aiiso Yufeng Li Family Dept Chem & Nano Engn, La Jolla, CA 92093 USA
[2] Univ Calif San Diego, Dept Pediat, Div Host Microbe Syst & Therapeut, La Jolla, CA 92093 USA
基金
美国国家卫生研究院;
关键词
CRISPR; in vivo gene editing; nanocarriers; recombinant adeno-associated viruses (rAAVs); virus-like particle (VLP); lipid nanoparticle (LNP); ADENOASSOCIATED VIRUS VECTOR; MESSENGER-RNA DELIVERY; LIPID NANOPARTICLES; ADENOVIRUS VECTORS; GUIDE-RNA; CATIONIC LIPIDS; CHEMICAL-MODIFICATIONS; GENERATION ADENOVIRUS; LENTIVIRAL VECTOR; SKELETAL-MUSCLE;
D O I
10.1007/s12274-024-6748-5
中图分类号
O64 [物理化学(理论化学)、化学物理学];
学科分类号
070304 ; 081704 ;
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.
引用
收藏
页码:8904 / 8925
页数:22
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