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Translational potential of base-editing tools for gene therapy of monogenic diseases. / Reshetnikov, Vasiliy V.; Chirinskaite, Angelina V.; Sopova, Julia V.; Ivanov, Roman A.; Leonova, Elena I.

в: Frontiers in Bioengineering and Biotechnology, Том 10, 942440, 10.08.2022.

Результаты исследований: Научные публикации в периодических изданиях › Обзорная статья › Рецензирование

Harvard

Reshetnikov, VV, Chirinskaite, AV, Sopova, JV, Ivanov, RA & Leonova, EI 2022, 'Translational potential of base-editing tools for gene therapy of monogenic diseases', Frontiers in Bioengineering and Biotechnology, Том. 10, 942440. https://doi.org/10.3389/fbioe.2022.942440

APA

Reshetnikov, V. V., Chirinskaite, A. V., Sopova, J. V., Ivanov, R. A., & Leonova, E. I. (2022). Translational potential of base-editing tools for gene therapy of monogenic diseases. Frontiers in Bioengineering and Biotechnology, 10, [942440]. https://doi.org/10.3389/fbioe.2022.942440

Vancouver

Reshetnikov VV, Chirinskaite AV, Sopova JV, Ivanov RA, Leonova EI. Translational potential of base-editing tools for gene therapy of monogenic diseases. Frontiers in Bioengineering and Biotechnology. 2022 Авг. 10;10. 942440. https://doi.org/10.3389/fbioe.2022.942440

Author

Reshetnikov, Vasiliy V. ; Chirinskaite, Angelina V. ; Sopova, Julia V. ; Ivanov, Roman A. ; Leonova, Elena I. / Translational potential of base-editing tools for gene therapy of monogenic diseases. в: Frontiers in Bioengineering and Biotechnology. 2022 ; Том 10.

BibTeX

@article{57abcd4dde2747a1b7fceef2866216a1,
title = "Translational potential of base-editing tools for gene therapy of monogenic diseases",
abstract = "Millions of people worldwide have rare genetic diseases that are caused by various mutations in DNA sequence. Classic treatments of rare genetic diseases are often ineffective, and therefore great hopes are placed on gene-editing methods. A DNA base–editing system based on nCas9 (Cas9 with a nickase activity) or dCas9 (a catalytically inactive DNA-targeting Cas9 enzyme) enables editing without double-strand breaks. These tools are constantly being improved, which increases their potential usefulness for therapies. In this review, we describe the main types of base-editing systems and their application to the treatment of monogenic diseases in experiments in vitro and in vivo. Additionally, to understand the therapeutic potential of these systems, the advantages and disadvantages of base-editing systems are examined.",
keywords = "base editing, dCas9, gene therapy, monogenic disease, nCas9, prime editor",
author = "Reshetnikov, {Vasiliy V.} and Chirinskaite, {Angelina V.} and Sopova, {Julia V.} and Ivanov, {Roman A.} and Leonova, {Elena I.}",
note = "Publisher Copyright: Copyright {\textcopyright} 2022 Reshetnikov, Chirinskaite, Sopova, Ivanov and Leonova.",
year = "2022",
month = aug,
day = "10",
doi = "10.3389/fbioe.2022.942440",
language = "English",
volume = "10",
journal = "Frontiers in Bioengineering and Biotechnology",
issn = "2296-4185",
publisher = "Baishideng Publishing Group",

}

RIS

TY - JOUR

T1 - Translational potential of base-editing tools for gene therapy of monogenic diseases

AU - Reshetnikov, Vasiliy V.

AU - Chirinskaite, Angelina V.

AU - Sopova, Julia V.

AU - Ivanov, Roman A.

AU - Leonova, Elena I.

N1 - Publisher Copyright: Copyright © 2022 Reshetnikov, Chirinskaite, Sopova, Ivanov and Leonova.

PY - 2022/8/10

Y1 - 2022/8/10

N2 - Millions of people worldwide have rare genetic diseases that are caused by various mutations in DNA sequence. Classic treatments of rare genetic diseases are often ineffective, and therefore great hopes are placed on gene-editing methods. A DNA base–editing system based on nCas9 (Cas9 with a nickase activity) or dCas9 (a catalytically inactive DNA-targeting Cas9 enzyme) enables editing without double-strand breaks. These tools are constantly being improved, which increases their potential usefulness for therapies. In this review, we describe the main types of base-editing systems and their application to the treatment of monogenic diseases in experiments in vitro and in vivo. Additionally, to understand the therapeutic potential of these systems, the advantages and disadvantages of base-editing systems are examined.

AB - Millions of people worldwide have rare genetic diseases that are caused by various mutations in DNA sequence. Classic treatments of rare genetic diseases are often ineffective, and therefore great hopes are placed on gene-editing methods. A DNA base–editing system based on nCas9 (Cas9 with a nickase activity) or dCas9 (a catalytically inactive DNA-targeting Cas9 enzyme) enables editing without double-strand breaks. These tools are constantly being improved, which increases their potential usefulness for therapies. In this review, we describe the main types of base-editing systems and their application to the treatment of monogenic diseases in experiments in vitro and in vivo. Additionally, to understand the therapeutic potential of these systems, the advantages and disadvantages of base-editing systems are examined.

KW - base editing

KW - dCas9

KW - gene therapy

KW - monogenic disease

KW - nCas9

KW - prime editor

UR - http://www.scopus.com/inward/record.url?scp=85136591211&partnerID=8YFLogxK

UR - https://www.mendeley.com/catalogue/3d2f11b1-bb64-3205-bb19-63857f08225b/

U2 - 10.3389/fbioe.2022.942440

DO - 10.3389/fbioe.2022.942440

M3 - Review article

AN - SCOPUS:85136591211

VL - 10

JO - Frontiers in Bioengineering and Biotechnology

JF - Frontiers in Bioengineering and Biotechnology

SN - 2296-4185

M1 - 942440

ER -

ID: 98530564