摘要
最近,基于 CRISPR 的技术显着提高了我们在各种基因组中进行所需更改和调节的能力。 其中,靶向碱基编辑是进行精确基因组编辑中最强大的技术之一。 碱基编辑使特定单个 DNA 碱基在所需基因组位点中从 C 到 T 或从 A 到 G 的不可逆转换成为可能。 考虑到其中许多是由点突变引起的,这项技术在人类遗传疾病的研究中具有重要意义。 更重要的是,这些编辑工具的高效率也为临床应用提供了广阔的前景。 在这篇综述中,我们讨论了碱基编辑工具的最新进展和挑战。
关键词: 基于 CRISPR 的技术、基因组、靶向碱基编辑、人类遗传疾病、点突变、碱基编辑工具。
Current Gene Therapy
Title:Recent Advances in CRISPR/Cas9 Directed Base Editing
Volume: 21 Issue: 4
关键词: 基于 CRISPR 的技术、基因组、靶向碱基编辑、人类遗传疾病、点突变、碱基编辑工具。
摘要: 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:
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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