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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...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
De Gruyter
2023
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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. |
format | Online Article Text |
id | pubmed-10471085 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | De Gruyter |
record_format | MEDLINE/PubMed |
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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