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单碱基编辑工具及在基因治疗中的应用及前景

陶永 赵幸乐 康文 吴皓

中华耳科学杂志2018,Vol.16Issue(2):150-154,5.
中华耳科学杂志2018,Vol.16Issue(2):150-154,5.DOI:10.3969/j.issn.1672-2922.2018.02.005

单碱基编辑工具及在基因治疗中的应用及前景

Base Editing and its Applications in Gene Therapy

陶永 1赵幸乐 1康文 1吴皓1

作者信息

  • 1. 上海交通大学医学院附属第九人民医院耳鼻咽喉头颈外科,上海交通大学医学院耳科学研究所,上海市耳鼻疾病转化医学重点实验室
  • 折叠

摘要

Abstract

The improvement of CRISPR/Cas9 gene editing efficiency has promoted its applications in bench and clinic research. However,the efficiency of CRISPR/Cas9 is not sufficient for gene modification.Base editing can con-vert one base pair to another permanently and efficiently,without double strains break.Although base editing was only recently developed,it has gained widespread utilization in gene editing,including in mouse inner ear models.Base edit-ing is an effective tool for inner ear gene therapy.

关键词

基因编辑/单碱基编辑/基因治疗

Key words

Gene Editing/Base Editing/Gene therapy

分类

医药卫生

引用本文复制引用

陶永,赵幸乐,康文,吴皓..单碱基编辑工具及在基因治疗中的应用及前景[J].中华耳科学杂志,2018,16(2):150-154,5.

基金项目

国家自然基金重点项目(81330023) (81330023)

国家自然基金面上项目(81700899) This work was supported by the State Key Program of National Natural Science Foundation of China(No.81330023).the National Natural Science Foundation of China(No.81700899). (81700899)

中华耳科学杂志

OA北大核心CSCDCSTPCD

1672-2922

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