Treatment of autosomal recessive hearing loss via in vivo CRISPR/Cas9-mediated optimized homology-directed repair in mice  

作　　者：Xi Gu Xinde Hu Daqi Wang Zhijiao Xu Fang Wang Di Li Geng-lin Li Hui Yang Huawei Li Erwei Zuo Yilai Shu 

机构地区：[1]ENT institute and Department of Otorhinolaryngology,Eye&ENT Hospital,State Key Laboratory of Medical Neurobiology and MOE Frontiers Center for Brain Science,Fudan University,Shanghai,China [2]Institutes of Biomedical Sciences,Fudan University,Shanghai,China [3]Department of Otolaryngology,the First Affiliated Hospital of Fujian Medical University,Fuzhou,China [4]NHC Key Laboratory of Hearing Medicine(Fudan University),Shanghai,China [5]Institute of Neuroscience,State Key Laboratory of Neuroscience,Key Laboratory of Primate Neurobiology,CAS Center for Excellence in Brain Science and Intelligence Technology,Shanghai Research Center for Brain Science and Brain-Inspired Intelligence,Shanghai Institutes for Biological Sciences,Chinese Academy of Sciences,Shanghai,China [6]State Key Lab for Conservation and Utilization of Subtropical Agric-Biological Resources,Guangxi University,Nanning,China [7]Shenzhen Branch,Guangdong Laboratory for Lingnan Modern Agriculture,Genome Analysis Laboratory of the Ministry of Agriculture,Agricultural Genomics Institute at Shenzhen,Chinese Academy of Agricultural Sciences,Shenzhen,China [8]The Institutes of Brain Science and the Collaborative Innovation Center for Brain Science,Fudan University,Shanghai,China

出　　处：《Cell Research》2022年第7期699-702,共4页细胞研究（英文版）

基　　金：supported by grants from the National Key R&D Program of China(2020YFA0908201,2017YFA0103900);the National Natural Science Foundation of China(82171148,81822011,31922048,81830029,82171141,82000992);the Science and Technology Commission of Shanghai Municipality(21S11905100,21JC401000);Clinic Research Plan of SHDC(SHDC2020CR4083),"Shuguang Program Hsupported by Shanghai Education Development Foundation and Shanghai Municipal Education Commission(20SG08),and the Agricultural Science and Technology Innovation Program(to E.Z.).

摘　　要：Dear Editor,Hearing loss is the most common sensory disorder in the world.Among cases of non-syndromic hearing loss,which account for 70%of all cases of genetic hearing loss,around 80%of cases arise from autosomal recessive loss-of-function mutations that require repair,rather than disruption,of the mutant allele.1 CRISPR/Cas9-mediated homology-directed repair(HDR)-based therapies have the potential to cure many genetic diseases because this class of therapeutics can achieve arbitrary base changes as well as the insertion or deletion of DNA fragments/However.

关 键 词：CRISPR/Cas9 directed HOMOLOGY 

分 类 号：R764[医药卫生—耳鼻咽喉科]