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Recent Advances in CRISPR/Cas9 Delivery Strategies

Yip, Bon·2020·Biomolecules
DOI 10.3390/biom10060839PMC PMC7356196
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Abstract

The clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system has revolutionized the field of gene editing. Continuous efforts in developing this technology have enabled efficient in vitro, ex vivo, and in vivo gene editing through a variety of delivery strategies. Viral vectors are commonly used in in vitro, ex vivo, and in vivo delivery systems, but they can cause insertional mutagenesis, have limited cloning capacity, and/or elicit immunologic responses. Physical delivery methods are largely restricted to in vitro and ex vivo systems, whereas chemical delivery methods require extensive optimization to improve their efficiency for in vivo gene editing. Achieving a safe and efficient in vivo delivery system for CRISPR/Cas9 remains the most challenging aspect of gene editing. Recently, extracellular vesicle-based systems were reported in various studies to deliver Cas9 in vitro and in vivo. In comparison with other methods, extracellular vesicles offer a safe, transient, and cost-effective yet efficient platform for delivery, indicating their potential for Cas9 delivery in clinical trials. In this review, we first discuss the pros and cons of different Cas9 delivery strategies. We then specifically review the development of extracellular vesicle-mediated gene editing and highlight the strengths and weaknesses of this technology.

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Metadata

Authors
Yip, B.
Affiliations
Vector Development and Production Laboratory, St. Jude Children’s Research Hospital, Memphis, TN 38105, USA
ORCID
Yip, Bon
Venue
Biomolecules
Publisher
MDPI AG
Published
2020-05-30
Volume
10
Issue
6
Pages
839
ISSN
2218-273X
DOI
10.3390/biom10060839
PMC
PMC7356196
Cited by
292
References
29
Language
en
Licence
creativecommons.org/licenses/by/4.0/ (vor)

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