Issue 13, 2022

Pre-clinical non-viral vectors exploited for in vivo CRISPR/Cas9 gene editing: an overview

Abstract

Clustered regulatory interspaced short palindromic repeats or CRISPR/Cas9 has emerged as a potent and versatile tool for efficient genome editing. This technology has been exploited for several applications including disease modelling, cell therapy, diagnosis, and treatment of many diseases including cancer. The in vivo application of CRISPR/Cas9 is hindered by poor stability, pharmacokinetic profile, and the limited ability of the CRISPR payloads to cross biological barriers. Although viral vectors have been implemented as delivery tools for efficient in vivo gene editing, their application is associated with high immunogenicity and toxicity, limiting their clinical translation. Hence, there is a need to explore new delivery methods that can guarantee safe and efficient delivery of the CRISPR/Cas9 components to target cells. In this review, we first provide a brief history and principles of nuclease-mediated gene editing, we then focus on the different CRISPR/Cas9 formats outlining their potentials and limitations. Finally, we discuss the alternative non-viral delivery strategies currently adopted for in vivo CRISPR/Cas9 gene editing.

Graphical abstract: Pre-clinical non-viral vectors exploited for in vivo CRISPR/Cas9 gene editing: an overview

Article information

Article type
Review Article
Submitted
16 9 2021
Accepted
13 4 2022
First published
23 5 2022
This article is Open Access
Creative Commons BY license

Biomater. Sci., 2022,10, 3410-3432

Pre-clinical non-viral vectors exploited for in vivo CRISPR/Cas9 gene editing: an overview

N. Rouatbi, T. McGlynn and K. T. Al-Jamal, Biomater. Sci., 2022, 10, 3410 DOI: 10.1039/D1BM01452H

This article is licensed under a Creative Commons Attribution 3.0 Unported Licence. You can use material from this article in other publications without requesting further permissions from the RSC, provided that the correct acknowledgement is given.

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