Yunnan Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming, Yunnan 650500, China.
Faculty of Life Science and Technology, Kunming University of Science and Technology, Kunming, Yunnan 650500, China.
Biomed Res Int. 2020 Aug 14;2020:2907623. doi: 10.1155/2020/2907623. eCollection 2020.
Using a base editor to generate monogenic disease models and correct pathogenic point mutations is a breakthrough technology for exploration and treatment of human diseases. As a burgeoning approach for genomic modification, the fused CRISPR/Cas9 with various deaminase separately has significantly increased the efficiency of producing a precise point mutation with minimal insertions or deletions (indels). Along with the flexibility and efficiency, a base editor has been widely used in many fields. This review discusses the recent development of a base editor, including evolution and advance, and highlights the applications and challenges in the field of gene therapy. Depending on rapid improvement and optimization of gene editing technology, the prospect of base editor is immeasurable.
利用碱基编辑器生成单基因疾病模型并纠正致病点突变是探索和治疗人类疾病的一项突破性技术。作为一种新兴的基因组修饰方法，融合 CRISPR/Cas9 与各种脱氨酶分别显著提高了产生精确点突变的效率，同时最小化插入或缺失（indels）的数量。碱基编辑器具有灵活性和高效性，已被广泛应用于许多领域。本文讨论了碱基编辑器的最新发展，包括其进化和进展，并强调了其在基因治疗领域的应用和挑战。随着基因编辑技术的快速改进和优化，碱基编辑器的前景不可估量。
