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Review Article

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

Zhongyuan Guo1,§, Audrey T. Zhu1,§, Ronnie H. Fang1,2( ), Liangfang Zhang1( )
Aiiso Yufeng Li Family Department of Chemical and Nano Engineering, University of California San Diego, La Jolla, CA 92093, USA
Division of Host-Microbe Systems and Therapeutics, Department of Pediatrics, University of California San Diego, La Jolla, CA 92093, USA

§ Zhongyuan Guo and Audrey T. Zhu contributed equally to this work.

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Abstract

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.

Graphical Abstract

The use of nanocarriers, including viral vectors, virus-like particles (VLPs), and lipid nanoparticles (LNPs), for the in vivo delivery of clustered regularly interspaced short palindromic repeats (CRISPR) gene editing systems is discussed in this review article.

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Nano Research
Pages 8904-8925

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Cite this article:
Guo Z, Zhu AT, Fang RH, et al. Viral and nonviral nanocarriers for in vivo CRISPR-based gene editing. Nano Research, 2024, 17(10): 8904-8925. https://doi.org/10.1007/s12274-024-6748-5
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Received: 18 March 2024
Revised: 08 May 2024
Accepted: 11 May 2024
Published: 20 June 2024
© Tsinghua University Press 2024