Abstract
Recently, CRISPR-based techniques have significantly improved our ability to make desired changes and regulations in various genomes. Among them, targeted base editing is one of the most powerful techniques in making precise genomic editing. Base editing enabled the irreversible conversion of a specific single DNA base, from C to T or and from A to G, in desired genomic loci. This technique has important implications in the study of human genetic diseases, considering that many of them resulted from point mutations. More importantly, the high efficiency of these editing tools also provided great promise in clinical applications. In this review, we discuss the recent progress and challenges of base editing tools.
Keywords: CRISPR based techniques, genomes, targeted base editing, human genetic diseases, point mutations, base editing tools.
Graphical Abstract
Current Gene Therapy
Title:Recent Advances in CRISPR/Cas9 Directed Base Editing
Volume: 21 Issue: 4
Author(s): Nan Liu, Lifang Zhou, Junyan Qu and Shaohua Yao*
Affiliation:
- Laboratory of Biotherapy, National Key Laboratory of Biotherapy, Cancer Center, West China Hospital, Sichuan University, Renmin Nanlu 17, Chengdu 610041, Sichuan,China
Keywords: CRISPR based techniques, genomes, targeted base editing, human genetic diseases, point mutations, base editing tools.
Abstract: Recently, CRISPR-based techniques have significantly improved our ability to make desired changes and regulations in various genomes. Among them, targeted base editing is one of the most powerful techniques in making precise genomic editing. Base editing enabled the irreversible conversion of a specific single DNA base, from C to T or and from A to G, in desired genomic loci. This technique has important implications in the study of human genetic diseases, considering that many of them resulted from point mutations. More importantly, the high efficiency of these editing tools also provided great promise in clinical applications. In this review, we discuss the recent progress and challenges of base editing tools.
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Cite this article as:
Liu Nan , Zhou Lifang , Qu Junyan and Yao Shaohua *, Recent Advances in CRISPR/Cas9 Directed Base Editing, Current Gene Therapy 2021; 21 (4) . https://dx.doi.org/10.2174/1566523221666210322090638
DOI https://dx.doi.org/10.2174/1566523221666210322090638 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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