发明名称 |
TREATMENT OF SYNUCLEINOPATHIES |
摘要 |
This invention relates generally to treating synucleinopathies in subjects that are not clinically diagnosed with a lysosomal storage disease, as well as associated methods of making medicaments and screening methods. |
申请公布号 |
US2016058849(A1) |
申请公布日期 |
2016.03.03 |
申请号 |
US201514820649 |
申请日期 |
2015.08.07 |
申请人 |
The Brigham and Women's Hospital, Inc. |
发明人 |
Schlossmacher Michael;Shihabuddin Lamya;Cheng Seng H.;Cullen Valerie |
分类号 |
A61K38/48;A61K45/06 |
主分类号 |
A61K38/48 |
代理机构 |
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代理人 |
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主权项 |
1. A method of treating a subject with a synucleinopathy, but not a clinically diagnosed lysosomal storage disease, the method comprising administering to a subject any one or more of:
a cathepsin F polypeptide, a procathepsin F polypeptide, and a polynucleotide encoding a cathepsin F or procathepsin F polypeptide, in an amount effective to reduce a level of α-synuclein in the subject's nervous system or in the subject's lysosomal compartment. |
地址 |
Boston MA US |