首页|单碱基编辑技术在治疗遗传性贫血中的应用

单碱基编辑技术在治疗遗传性贫血中的应用

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单碱基编辑器(base editors,BEs)是一类基于CRISPR/Cas系统或转录激活子样效应子阵列蛋白(transcription activator-like effector,TALE)基因编辑技术的新兴碱基编辑器。该类编辑器既不需要引入DNA双键断裂,也不需要供体DNA,可针对基因组单个碱基进行精准替换,与以前的基因编辑技术相比,单碱基编辑器具有副产物更少、碱基编辑范围更大等优点,这为基因治疗点突变导致的遗传性贫血带来了曙光。本文对单碱基编辑技术的发展,以及单碱基编辑技术在基因治疗突变引起的遗传性贫血的临床尝试中取得的进展进行概述,可为未来临床上基因治疗遗传性贫血提供理论参考。
Applications of the Single Base Editing Technology in the Treatment of Hereditary Anemia
Single base editors(BEs)are a new class of base editors derived from the CRISPR/Cas sys-tem or transcription activator-like effector(TALE)gene editing technology,which can accurately edit a single base without introducing DNA double-strand-breaks or donor DNA.Compared with the previous genome editing technologies,it has the advantages of less by-products,higher editing efficiency,and lar-ger base editing range,which can be used for the treatment of genetic anemia caused by point mutations.In this paper,the development of single base editing technology and its progress in gene therapy for trea-ting genetic anemia caused by gene mutations are summarized,which can provide a theoretical reference for the clinical trial of gene therapy for clinical anemia.

single base editor(BEs)hereditary anemiagene mutation

王丽媛、付瑜瑜、谢元斌

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赣南医科大学基础医学院,江西赣州 341000

宝鸡市中医医院,陕西宝鸡 721000

单碱基编辑器 遗传性贫血 基因突变

国家自然科学基金项目江西省自然科学基金项目

3216018920202BABL216070

2024

中国生物化学与分子生物学报
中国生物化学与分子生物学会 北京大学

中国生物化学与分子生物学报

CSTPCD北大核心
影响因子:0.617
ISSN:1007-7626
年,卷(期):2024.40(6)
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