Nonviral Delivery of CRISPR/Cas Systems in mRNA Format. Issue 11 (15th September 2022)

• Record Type: Journal Article
• Authors: Liu, Zhen; Li, Zhenghua; Li, Bin
• Is Part Of: Advanced nanobiomed research. Volume 2:Issue 11(2022)
• Page Start: n/a
• Page End: n/a
• Publication Date: 2022-09-15
• Subjects: clustered regularly interspaced short palindromic repeat (CRISPR)/Cas system -- mRNA -- nanomaterials -- nonviral delivery; Nanomedicine -- Periodicals; Biomedical engineering -- Periodicals; Biomedical materials -- Periodicals; Nanomedicine; Nanostructures; Bioengineering; Biocompatible Materials; Electronic journals; Periodicals; Periodical; 610.28
• DOI: 10.1002/anbr.202200082
• Languages: English
• ISSNs: 2699-9307

Abstract

Abstract : The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR‐associated (Cas) genome editing system provides a powerful toolbox for genetic engineering. However, safe and efficient in vivo delivery of all CRISPR components into the nuclei of target organs remains challenging toward CRISPR/Cas clinical translation. With the rapid development of nanotechnology, the nonviral CRISPR/Cas mRNA delivery systems are emerging as one of the most clinically advanced platforms. CRISPR/Cas mRNA formulated in lipid nanoparticles is currently under clinical trials for treating hereditary transthyretin amyloidosis. Nonviral delivery of CRISPR/Cas systems in mRNA format not only minimizes immunogenicity and carcinogenicity, but also enables transient genome editing, thereby reducing potential off‐target effects. The recent advances in the nonviral CRISPR/Cas mRNA delivery systems should facilitate CRISPR technologies toward clinical applications. Abstract : Nonviral vectors hold promise for delivery of clustered regularly interspaced short palindromic repeats (CRISPR)/Cas systems in mRNA format. This review summarizes the recent advances in the nonviral CRISPR/Cas mRNA delivery systems and their applications in various diseases, emphasizing the biodistribution, gene editing efficiency, and therapeutic effects in vitro and in vivo.