Issue 5, 2022

Non-viral delivery of the CRISPR/Cas system: DNA versus RNA versus RNP

Abstract

Since its discovery, the CRISPR/Cas technology has rapidly become an essential tool in modern biomedical research. The opportunities to specifically modify and correct genomic DNA have also raised big hope for therapeutic applications by direct in vivo genome editing. In order to achieve the intended genome modifications, the functional unit of the CRISPR/Cas system finally has to be present in the nucleus of target cells. This can be achieved by delivery of different biomolecular Cas9 and gRNA formats: plasmid DNA (pDNA), RNA or Cas9 ribonucleoproteins (RNPs). While the initial research focussed on pDNA transfections, the currently most promising strategy for systemic non-viral in vivo delivery is based on RNA which has achieved remarkable results in the first clinical trials. RNP delivery receives much attention for ex vivo applications, but the translation to systemic in vivo genome editing in patients has not been reached so far. The article summarises the characteristics and differences of each format, provides an overview of the published delivery strategies and highlights recent examples of delivery systems including the status of clinical applications.

Graphical abstract: Non-viral delivery of the CRISPR/Cas system: DNA versus RNA versus RNP

Supplementary files

Article information

Article type
Review Article
Submitted
29 اکتوٗبر 2021
Accepted
24 جنؤری 2022
First published
25 جنؤری 2022
This article is Open Access
Creative Commons BY license

Biomater. Sci., 2022,10, 1166-1192

Non-viral delivery of the CRISPR/Cas system: DNA versus RNA versus RNP

Y. Lin, E. Wagner and U. Lächelt, Biomater. Sci., 2022, 10, 1166 DOI: 10.1039/D1BM01658J

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