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CRISPR/Cas9: Principle, Applications, and Delivery through Extracellular Vesicles

Horodecka, Katarzyna, Düchler, Markus·2021·International Journal of Molecular Sciences

Abstract

The establishment of CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) technology for eukaryotic gene editing opened up new avenues not only for the analysis of gene function but also for therapeutic interventions. While the original methodology allowed for targeted gene disruption, recent technological advancements yielded a rich assortment of tools to modify genes and gene expression in various ways. Currently, clinical applications of this technology fell short of expectations mainly due to problems with the efficient and safe delivery of CRISPR/Cas9 components to living organisms. The targeted in vivo delivery of therapeutic nucleic acids and proteins remain technically challenging and further limitations emerge, for instance, by unwanted off-target effects, immune reactions, toxicity, or rapid degradation of the transfer vehicles. One approach that might overcome many of these limitations employs extracellular vesicles as intercellular delivery devices. In this review, we first introduce the CRISPR/Cas9 system and its latest advancements, outline major applications, and summarize the current state of the art technology using exosomes or microvesicles for transporting CRISPR/Cas9 constituents into eukaryotic cells.

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Metadata

Authors
Horodecka, K., & Düchler, M.
Affiliations
Department of Bioorganic Chemistry, Centre of Molecular and Macromolecular Studies, Polish Academy of Sciences, 112 Sienkiewicza Street, 90-363 Lodz, Poland
Venue
International Journal of Molecular Sciences
Publisher
MDPI AG
Published
2021-06-04
Volume
22
Issue
11
Pages
6072
ISSN
1422-0067
Cited by
121
References
18
Language
en
Funders
Narodowym Centrum Nauki (UMO-2019/35/B/NZ7/03256)

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