CRISPR-Cas9 base editors and their current role in human therapeutics

Walker S. Lahr; Christopher J. Sipe; Joseph G. Skeate; Beau R. Webber; Branden S. Moriarity

doi:10.1016/j.jcyt.2022.11.013

CRISPR-Cas9 base editors and their current role in human therapeutics

Walker S. Lahr, Christopher J. Sipe, Joseph G. Skeate, Beau R. Webber, Branden S. Moriarity

Pediatrics - Hematology

Research output: Contribution to journal › Article › peer-review

6 Scopus citations

Abstract

Background: Consistent progress has been made to create more efficient and useful CRISPR-Cas9-based molecular toolsfor genomic modification. Methods: This review focuses on recent articles that have employed base editors (BEs) for both clinical and research purposes. Results: CRISPR-Cas9 BEs are a useful system because of their highefficiency and broad applicability to gene correction and disruption. In addition, base editing has beensuggested as a safer approach than other CRISPR-Cas9-based systems, as it limits double-strand breaksduring multiplex gene knockout and does not require a toxic DNA donor molecule for genetic correction. Conclusion: As such, numerous industry and academic groups are currently developing base editing strategies withclinical applications in cancer immunotherapy and gene therapy, which this review will discuss, with a focuson current and future applications of in vivo BE delivery.

Original language	English (US)
Pages (from-to)	270-276
Number of pages	7
Journal	Cytotherapy
Volume	25
Issue number	3
DOIs	https://doi.org/10.1016/j.jcyt.2022.11.013
State	Published - Mar 2023

Bibliographical note

Publisher Copyright:
© 2022

Keywords

base editor
cancer immunotherapy
gene therapy
hematopoietic stem cell
multiplex gene editing
sickle cell

PubMed: MeSH publication types

Review
Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1016/j.jcyt.2022.11.013

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title = "CRISPR-Cas9 base editors and their current role in human therapeutics",

abstract = "Background: Consistent progress has been made to create more efficient and useful CRISPR-Cas9-based molecular toolsfor genomic modification. Methods: This review focuses on recent articles that have employed base editors (BEs) for both clinical and research purposes. Results: CRISPR-Cas9 BEs are a useful system because of their highefficiency and broad applicability to gene correction and disruption. In addition, base editing has beensuggested as a safer approach than other CRISPR-Cas9-based systems, as it limits double-strand breaksduring multiplex gene knockout and does not require a toxic DNA donor molecule for genetic correction. Conclusion: As such, numerous industry and academic groups are currently developing base editing strategies withclinical applications in cancer immunotherapy and gene therapy, which this review will discuss, with a focuson current and future applications of in vivo BE delivery.",

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AU - Sipe, Christopher J.

AU - Skeate, Joseph G.

AU - Webber, Beau R.

AU - Moriarity, Branden S.

PY - 2023/3

Y1 - 2023/3

N2 - Background: Consistent progress has been made to create more efficient and useful CRISPR-Cas9-based molecular toolsfor genomic modification. Methods: This review focuses on recent articles that have employed base editors (BEs) for both clinical and research purposes. Results: CRISPR-Cas9 BEs are a useful system because of their highefficiency and broad applicability to gene correction and disruption. In addition, base editing has beensuggested as a safer approach than other CRISPR-Cas9-based systems, as it limits double-strand breaksduring multiplex gene knockout and does not require a toxic DNA donor molecule for genetic correction. Conclusion: As such, numerous industry and academic groups are currently developing base editing strategies withclinical applications in cancer immunotherapy and gene therapy, which this review will discuss, with a focuson current and future applications of in vivo BE delivery.

AB - Background: Consistent progress has been made to create more efficient and useful CRISPR-Cas9-based molecular toolsfor genomic modification. Methods: This review focuses on recent articles that have employed base editors (BEs) for both clinical and research purposes. Results: CRISPR-Cas9 BEs are a useful system because of their highefficiency and broad applicability to gene correction and disruption. In addition, base editing has beensuggested as a safer approach than other CRISPR-Cas9-based systems, as it limits double-strand breaksduring multiplex gene knockout and does not require a toxic DNA donor molecule for genetic correction. Conclusion: As such, numerous industry and academic groups are currently developing base editing strategies withclinical applications in cancer immunotherapy and gene therapy, which this review will discuss, with a focuson current and future applications of in vivo BE delivery.

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KW - gene therapy

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KW - multiplex gene editing

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CRISPR-Cas9 base editors and their current role in human therapeutics

Abstract

Bibliographical note

Keywords

PubMed: MeSH publication types

UN SDGs

Access

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