公开(公告)号：WO2018039147A1

公开(公告)日：2018-03-01

申请号：PCT/US2017/047878

申请日：2017-08-22

Applicant: THE JOHNS HOPKINS UNIVERSITY ,  UNIVERSITY OF PITTSBURGH - OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION

Inventor： DAWSON, Ted M. ,  DAWSON, Valina L. ,  KO, Han Seok ,  MAO, Xiaobo ,  ALBERTO VIGNALI, Dario Angelo ,  WORKMAN, Creg J.

IPC: A61K38/00 ,  A61K48/00 ,  A61P25/16 ,  A61P25/28

CPC classification number: C07K16/2803 ,  A01K2217/052 ,  A01K2227/105 ,  A01K2267/0318 ,  A61K31/713 ,  A61P21/00 ,  A61P25/16 ,  A61P25/28 ,  C07K2317/76 ,  C12N15/1138 ,  C12N2310/11 ,  G01N33/573 ,  G01N33/6896 ,  G01N2500/02 ,  G01N2800/2835

Abstract: Described are methods of inhibiting neurodegeneration in a subject by administering to the subject an agent that prevents (alpha)-syn PFF from binding to its receptor. The agent may be a small molecule chemical compound, antibody, nucleic acid molecule, or polypeptide. Drug screening methods are also provided.

Abstract translation: 描述了抑制受试者神经变性的方法，其通过向受试者施用防止α-syn PFF与其受体结合的试剂。 试剂可以是小分子化合物，抗体，核酸分子或多肽。 还提供了药物筛选方法。

公开(公告)号：WO2011057204A2

公开(公告)日：2011-05-12

申请号：PCT/US2010/055857

申请日：2010-11-08

Applicant: THE JOHNS HOPKINS UNIVERSITY ,  LEE, Byoung Dae ,  DAWSON, Ted M. ,  DAWSON, Valina L. ,  WEST, Andrew B.

Inventor： LEE, Byoung Dae ,  DAWSON, Ted M. ,  DAWSON, Valina L. ,  WEST, Andrew B.

IPC: A61K31/403 ,  A61K31/416 ,  A61K31/404 ,  A61K31/519 ,  A61P25/28 ,  A61P25/00

CPC classification number: A61K31/553 ,  A61K31/122 ,  A61K31/403 ,  A61K31/404 ,  A61K31/416 ,  A61K31/519 ,  A61K31/7064

Abstract: Leucine-rich repeat kinase-2 (LRRK2) mutations are a common cause of Parkinson's disease. Inhibitors of LRRK2 kinase that are protective in in vitro and in vivo models of LRRK2-induced neurodegeneration were identified. The presently disclosed subject matter establishes that LRRK2-induced degeneration of neurons in vivo is kinase dependent and that LRRK2 kinase inhibition provides a potential new neuroprotective paradigm for treating Parkinson's disease.

Abstract translation: 亮氨酸重复激酶-2（LRRK2）突变是帕金森病的常见病因。 鉴定了在LRRK2诱导的神经变性的体外和体内模型中保护的LRRK2激酶的抑制剂。 目前公开的主题确定LRRK2诱导的体内神经元的变性是激酶依赖性的，并且LRRK2激酶抑制提供了治疗帕金森病的潜在的新的神经保护范例。

公开(公告)号：WO2018045250A1

公开(公告)日：2018-03-08

申请号：PCT/US2017/049778

申请日：2017-08-31

Applicant: THE JOHNS HOPKINS UNIVERSITY

Inventor： DAWSON, Ted M. ,  DAWSON, Valina L. ,  WANG, Yingfei ,  PARK, Hyejin ,  LIU, Jun ,  PENG, Hanjing

IPC: A61P25/00 ,  A61P25/28 ,  A61K38/17

Abstract: Provided herein are methods of treating a disease, such as Parkinson's disease, that is due to increased poly [ADP-ribose] polymerase 1 (PARP-1) activation, by inhibiting macrophage migration inhibitory factor (MIF) nuclease activity.

Abstract translation: 本文提供了治疗由于增加的聚[ADP-核糖]聚合酶1（PARP-1）激活，通过抑制巨噬细胞迁移抑制因子（MIF）引起的疾病（例如帕金森氏病）的方法 ）核酸酶活性。