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Precise genome editing with base editors

Single-nucleotide variants account for about half of known pathogenic genetic variants in human. Genome editing strategies by reversing pathogenic point mutations with minimum side effects have great therapeutic potential and are now being actively pursued. The emerge of precise and efficient genome...

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Detalles Bibliográficos
Autores principales: Liu, Hongcai, Zhu, Yao, Li, Minjie, Gu, Zhimin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: De Gruyter 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10471085/
https://www.ncbi.nlm.nih.gov/pubmed/37724105
http://dx.doi.org/10.1515/mr-2022-0044
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author Liu, Hongcai
Zhu, Yao
Li, Minjie
Gu, Zhimin
author_facet Liu, Hongcai
Zhu, Yao
Li, Minjie
Gu, Zhimin
author_sort Liu, Hongcai
collection PubMed
description Single-nucleotide variants account for about half of known pathogenic genetic variants in human. Genome editing strategies by reversing pathogenic point mutations with minimum side effects have great therapeutic potential and are now being actively pursued. The emerge of precise and efficient genome editing strategies such as base editing and prime editing provide powerful tools for nucleotide conversion without inducing double-stranded DNA breaks (DSBs), which have shown great potential for curing genetic disorders. A diverse toolkit of base editors has been developed to improve the editing efficiency and accuracy in different context of application. Here, we summarized the evolving of base editors (BEs), their limitations and future perspective of base editing-based therapeutic strategies.
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spelling pubmed-104710852023-09-18 Precise genome editing with base editors Liu, Hongcai Zhu, Yao Li, Minjie Gu, Zhimin Med Rev (Berl) Review Single-nucleotide variants account for about half of known pathogenic genetic variants in human. Genome editing strategies by reversing pathogenic point mutations with minimum side effects have great therapeutic potential and are now being actively pursued. The emerge of precise and efficient genome editing strategies such as base editing and prime editing provide powerful tools for nucleotide conversion without inducing double-stranded DNA breaks (DSBs), which have shown great potential for curing genetic disorders. A diverse toolkit of base editors has been developed to improve the editing efficiency and accuracy in different context of application. Here, we summarized the evolving of base editors (BEs), their limitations and future perspective of base editing-based therapeutic strategies. De Gruyter 2023-02-22 /pmc/articles/PMC10471085/ /pubmed/37724105 http://dx.doi.org/10.1515/mr-2022-0044 Text en © 2023 the author(s), published by De Gruyter, Berlin/Boston https://creativecommons.org/licenses/by-nc-nd/4.0/This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License.
spellingShingle Review
Liu, Hongcai
Zhu, Yao
Li, Minjie
Gu, Zhimin
Precise genome editing with base editors
title Precise genome editing with base editors
title_full Precise genome editing with base editors
title_fullStr Precise genome editing with base editors
title_full_unstemmed Precise genome editing with base editors
title_short Precise genome editing with base editors
title_sort precise genome editing with base editors
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10471085/
https://www.ncbi.nlm.nih.gov/pubmed/37724105
http://dx.doi.org/10.1515/mr-2022-0044
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