公开(公告)号：EP2831232A1

公开(公告)日：2015-02-04

申请号：EP13770075.3

申请日：2013-03-14

申请人： Washington University ,  Isis Pharmaceuticals, Inc.

发明人： MILLER, Timothy M. ,  DEVOS, Sarah ,  BENNETT, C. Frank ,  RIGO, Frank

IPC分类号： C12N15/11 ,  C07H21/00

CPC分类号： C12N15/113 ,  C07H21/02 ,  C07H21/04 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3525

摘要： Disclosed herein are methods for reducing expression of Tau mRNA and protein in an animal with Tau antisense compounds. Also disclosed are methods for modulating splicing of Tau mRNA in an animal with Tau antisense compounds. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration Tau antisense oligonucleotides include Alzheimer's Disease, Fronto-temporal Dementia (FTD), FTDP-17, Progressive Supranuclear Palsy, Chronic Traumatic Encephalopathy, Epilepsy, and Dravet's Syndrome.

摘要翻译： 本文公开了用Tau反义化合物降低动物中Tau mRNA和蛋白质表达的方法。 还公开了用Tau反义化合物调节动物中Tau mRNA的剪接的方法。 此类方法可用于治疗，预防或改善有需要的个体中的神经变性疾病。 可以通过施用Tau反义寡核苷酸治疗，预防和改善的神经变性疾病的实例包括阿尔茨海默氏病，额颞叶痴呆（FTD），FTDP-17，进行性核上性麻痹，慢性创伤性脑病，癫痫和Dravet综合征。

公开(公告)号：EP2769212A1

公开(公告)日：2014-08-27

申请号：EP12853146.4

申请日：2012-10-17

申请人： The Washington University ,  Bateman, Randall J. ,  Miller, Timothy M. ,  Munsell, Ling

发明人： BATEMAN, Randall J. ,  MILLER, Timothy M. ,  MUNSELL, Ling

IPC分类号： G01N33/00 ,  B01D59/44 ,  G01N23/00

CPC分类号： G01N33/577 ,  C12Q1/26 ,  G01N33/5058 ,  G01N33/5088 ,  G01N33/6896 ,  G01N2333/90283 ,  G01N2560/00 ,  G01N2800/28

摘要： The disclosure relates to methods for the diagnosis and treatment of neurological and neurodegenerative diseases, disorders, and associated processes. Specifically, the disclosure relates to a method for measuring the metabolism of central nervous system derived biomolecules in a subject in vivo. Further disclosed are methods for measuring the in vivo metabolism (e.g. the rate of synthesis, the rate of clearance) of neurally derived biomolecules, such as superoxide dismutase 1 (SOD1 ).