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 代理人
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