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 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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