Stereopure AIMer:A Promising RNA Base-editing Tool for Monogenic Neurological Diseases  

作　　者：Jingyu Yu Tianwen Li Kezhu Chen Qisheng Tang Jianhong Zhu 

机构地区：[1]Department of Neurosurgery,Huashan Hospital,Shanghai Medical College,Fudan University,National Center for Neurological Disorders,National Key Laboratory for Medical Neurobiology,Institutes of Brain Science,Shanghai Key Laboratory of Brain Function and Regeneration,Institute of Neurosurgery,MOE Frontiers Center for Brain Science,Shanghai 200000,China

出　　处：《Neuroscience Bulletin》2023年第2期353-355,共3页神经科学通报（英文版）

基　　金：supported by grants from the Ministry of Science and Technology of China(2018YFA0107900,92168103,32171417,82001140,2019CXJQ01);the National Nature Science Foundation,and Shanghai Municipal Government,Peak Disciplines(TypeⅣ)of Institutions of Higher Leaning in Shanghai。

摘　　要：The continuous development of various gene therapies has brought new light to the treatment of genetic diseases.Among them,therapies targeting monogenic diseases are relatively progressive and promising due to the explicit and clear pathogenesis.The discovery and development of clustered regularly interspaced short palindromic repeats/associated nuclease(CRISPR/Cas)and related technologies was undoubtedly an extraordinary leap forward for gene therapy.

关 键 词：CRISPR/Cas STEREO DOUBT 

分 类 号：R741.02[医药卫生—神经病学与精神病学]