公开(公告)号：US20070185042A1

公开(公告)日：2007-08-09

申请号：US10567537

申请日：2004-08-09

申请人： Li-Huei Tsai ,  Kenneth Kosik

发明人： Li-Huei Tsai ,  Kenneth Kosik

IPC分类号： A61K48/00 ,  C07H21/02 ,  C12N5/08

CPC分类号： C12N15/1137 ,  A61K38/00 ,  C12N2310/111 ,  C12N2310/14

摘要： The present invention relates to siRNA molecules derived from BACE1 and BACE2 genes. Accordingly, provided herein are siRNA molecules comprising a nucleotide sequence consisting essentially of a BACE1 or BACE2 gene. Also provided are methods for reducing the level of BACE1 protein in a cell. Further provided are methods for preparing a pharmaceutical composition comprising an siRNA with a pharmaceutically acceptable carrier.

摘要翻译： 本发明涉及衍生自BACE1和BACE2基因的siRNA分子。 因此，本文提供了包含基本上由BACE1或BACE2基因组成的核苷酸序列的siRNA分子。 还提供了降低细胞中BACE1蛋白水平的方法。 还提供了制备包含具有药学上可接受的载体的siRNA的药物组合物的方法。

公开(公告)号：US20220127347A1

公开(公告)日：2022-04-28

申请号：US17511343

申请日：2021-10-26

申请人： Kenneth Kosik ,  Jennifer Rauch

发明人： Kenneth Kosik ,  Jennifer Rauch

IPC分类号： C07K16/18

摘要： Provided are novel methods of inhibiting the propagation of pathological forms of tau between cells of the central nervous system by administration of an inhibitor of LRP1. LRP1 was discovered to facilitate the endocytosis of pathological forms of tau which enables the propagation of tau aggregates and resulting neurodegeneration. These methods enable the prevention and treatment of various tauopathies, such as Alzheimer's, chronic traumatic encephalopathy, corticobasal degeneration, and others.