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Mitigating off-target effects in CRISPR/Cas9-mediated in vivo gene editing

Han, Hua Alexander, Pang, Jeremy Kah Sheng, Soh, Boon-Seng·2020·Journal of Molecular Medicine
DOI 10.1007/s00109-020-01893-zPMC PMC7220873
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Abstract

The rapid advancement of genome editing technologies has opened up new possibilities in the field of medicine. Nuclease-based techniques such as the CRISPR/Cas9 system are now used to target genetically linked disorders that were previously hard-to-treat. The CRISPR/Cas9 gene editing approach wields several advantages over its contemporary editing systems, notably in the ease of component design, implementation and the option of multiplex genome editing. While results from the early phase clinical trials have been encouraging, the small patient population recruited into these trials hinders a conclusive assessment on the safety aspects of the CRISPR/Cas9 therapy. Potential safety concerns include the lack of fidelity in the CRISPR/Cas9 system which may lead to unintended DNA modifications at non-targeted gene loci. This review focuses modifications to the CRISPR/Cas9 components that can mitigate off-target effects in in vitro and preclinical models and its translatability to gene therapy in patient populations.

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Fig. 1 Graphical outline of the review article and overview of the mitigating techniques that can be applied to minimise OTEs in CRISPR/Cas9 genome editing
Fig. 1 Graphical outline of the review article and overview of the mitigating techniques that can be applied to minimise OTEs in CRISPR/Cas9 genome editingCC BY · PubMed Central
Fig. 2 Stagewise schematic representations of target site recognition in CRISPR/Cas9-mediated genome editing with modifications to the sgRNA and Cas9 endonuclease to reduce OTEs. a The Cas9 endonuclease first scans the genomic DNA and binds to canonical PAM sequences (I). This induces a structural change in the sgRNA that allows the guide sequence to search and hybridise to complementary target sites upstream of the PAM (II). sgRNA-DNA hybridisation activates the Cas9 nuclease domains which then cleaves both strands of DNA (III). b sgRNA can be truncated at the 5′-end by 2–3 nucleotides or c modified at the 5′-end to contain 2 guanine nucleotides to improve the specificity of the guide sequence. d Cas9 nickase with only 1 active catalytic domain can be paired and e catalytically deactivated Cas9 fused to FokI nuclease that requires dimerisation for nuclease activity can be used to minimise off-target indels (insertion/deletion). f Base editors that convert a single cytosine base to thymine without requiring DSBs are less promiscuous at off-target sites. g Cas9 orthologs from other bacteria such as SaCas9 and h other Cas nucleases such as Cas12a that recognises alternative PAMs can be used to target novel DNA sequences and improve specificity
Fig. 2 Stagewise schematic representations of target site recognition in CRISPR/Cas9-mediated genome editing with modifications to the sgRNA and Cas9 endonuclease to reduce OTEs. a The Cas9 endonuclease first scans the genomic DNA and binds to canonical PAM sequences (I). This induces a structural change in the sgRNA that allows the guide sequence to search and hybridise to complementary target sites upstream of the PAM (II). sgRNA-DNA hybridisation activates the Cas9 nuclease domains which then cleaves both strands of DNA (III). b sgRNA can be truncated at the 5′-end by 2–3 nucleotides or c modified at the 5′-end to contain 2 guanine nucleotides to improve the specificity of the guide sequence. d Cas9 nickase with only 1 active catalytic domain can be paired and e catalytically deactivated Cas9 fused to FokI nuclease that requires dimerisation for nuclease activity can be used to minimise off-target indels (insertion/deletion). f Base editors that convert a single cytosine base to thymine without requiring DSBs are less promiscuous at off-target sites. g Cas9 orthologs from other bacteria such as SaCas9 and h other Cas nucleases such as Cas12a that recognises alternative PAMs can be used to target novel DNA sequences and improve specificityCC BY · PubMed Central

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Cited by

Sorted by citations

Metadata

Authors
Han, H. A., Pang, J. K. S., & Soh, B.-S.
Venue
Journal of Molecular Medicine
Publisher
Springer Science and Business Media LLC
Published
2020-03-20
Volume
98
Issue
5
Pages
615-632
ISSN
  • 0946-2716
  • 1432-1440
DOI
10.1007/s00109-020-01893-z
PMC
PMC7220873
Cited by
134
References
46
Language
en
Licence
Funders
  • National University of Singapore (Graduate scholarship)
  • National Medical Research Council (NMRC/OFYIRG/0017/2016)

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