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
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)