发明名称 TREATMENT OF SYNUCLEINOPATHIES
摘要 PROBLEM TO BE SOLVED: To provide a method of treating synucleinopathies in subjects that are not generally diagnosed clinically with a lysosomal storage disease, as well as associated methods of making medicaments and screening methods.SOLUTION: There is administered a pharmaceutical composition including an effective amount of one or both of acidic &bgr;-glucocerebrosidase (GBA) polypeptide and polynucleotide encoding the acidic &bgr;-glucocerebrosidase (GBA) polypeptide, for lowering a level of α-synuclein in an object nervous system or an object lysosome compartment.
申请公布号 JP2013241411(A) 申请公布日期 2013.12.05
申请号 JP20130119398 申请日期 2013.06.06
申请人 BRIGHAM & WOMEN'S HOSPITAL INC 发明人 SCHLOSSMACHER MICHAEL;CULLEN VALERIE;SHIHABUDDIN LAMYA;CHENG SENG H
分类号 A61K38/00;A61K31/047;A61K31/196;A61K31/436;A61K31/706;A61K31/7088;A61K39/395;A61K45/00;A61K48/00;A61P21/02;A61P25/00;A61P25/02;A61P25/16;A61P25/20;A61P25/28;A61P43/00 主分类号 A61K38/00
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