Inhibition of PI4KIII α as a Novel Potential Approach for Gaucher Disease Treatment  

作　　者：Linan Zheng Feng Hong Fude Huang Wenan Wang 

机构地区：[1]Department of Neurology,Xinhua Hospital Affiliated to Shanghai Jiaotong University School of Medicine,Shanghai,200092,China [2]Department of Neurology,Xinhua Hospital Chongming Branch,Shanghai,202150,China [3]Nuo-Beta Pharmaceutical Technology(Shanghai)Co.Ltd,Shanghai,201210,China [4]University of the Chinese Academy of Sciences,Sino-Danish College,Chinese Academy of Sciences,Beijing,100049,China

出　　处：《Neuroscience Bulletin》2021年第8期1234-1239,共6页神经科学通报（英文版）

基　　金：the National Natural Science Foundation of China(81771416,81650110527,and 8197100);Shanghai Municipal Commission of Health and Family Planning(201740153);Key Discipline of Chongming District,Shanghai,China,2018.

摘　　要：Dear Editor,Gaucher disease(GD)is the most common lysosomal storage disease(LSD)caused by an insufficiency of the lysosomal enzyme glucocerebrosidase(GCase)[1].GCase insufficiency produces the excessive lysosomal accumulation of unmetabolized glycolipid substrates including glucosylceramide(GlcCer),leading to the disruption of the structure and function of tissues and organs,includingthe blood system,viscera,brain,boncs,and cartilage.

关 键 词：INSUFFICIENCY ORGANS GAUCHER 

分 类 号：R741[医药卫生—神经病学与精神病学]