CRISPR base editing-mediated correction of a tau mutation rescues cognitive decline in a mouse model of tauopathy  

作　　者：Min Sung Gee Eunji Kwon Myeong-Hoon Song Seung Ho Jeon Namkwon Kim Jong Kil Lee Taeyoung Koo 

机构地区：[1]College of Pharmacy,Kyung Hee University,Seoul 02447,Republic of Korea [2]Department of Biomedical and Pharmaceutical Sciences,Graudate School,Kyung Hee University,Seoul 02447,Republic of Korea [3]Department of Pharmaceutical Sciences,College of Pharmacy,Kyung Hee University,Seoul 02447,Republic of Korea

出　　处：《Translational Neurodegeneration》2024年第1期828-832,共5页转化神经变性病(英文)

基　　金：supported by the National Research Foundation of Korea(NRF)grant funded by the Korea government(MSIT)(2022R1A2C1013352 and RS-2023–00262386 to T.K.);Ministry of Food and Drug Safety(21153MFDS601 to T.K.);the Medical Research Center Program through the National Research Foundation of Korea funded by the Ministry of Science and ICT(NRF-2017R1A5A2014768 to J.K.L.).

摘　　要：Main text The microtubule-binding protein tau is encoded by MAPT,located on chromosome 17.Mutations in this gene have been implicated in frontotemporal dementia[1].Down-regulation of endogenous tau with antisense oligonucleotides(ASOs)specific for human tau or zinc-finger protein transcription factors has been explored in preclinical models of tauopathy[2,3].Of particular note,the effects of tau ASOs on mild Alzheimer’s disease are now under assessment in a clinical trial[4].In addition,CRISPR-mediated gene knockout has been used to regu-late the expression of APP or BACE1 to ameliorate amy-loidβand tau pathologies[5,6].

关 键 词：TAU MEDIATED ENDOGENOUS 

分 类 号：R741[医药卫生—神经病学与精神病学]