Invention Grant
US09226935B2 Methods for modulating the expression and aggregation of a CAG-expanded gene product in cells and methods for identifying agents useful for doing the same
有权
用于调节细胞中CAG扩增的基因产物的表达和聚集的方法以及用于鉴定可用于进行相同的试剂的方法
- Patent Title: Methods for modulating the expression and aggregation of a CAG-expanded gene product in cells and methods for identifying agents useful for doing the same
- Patent Title (中): 用于调节细胞中CAG扩增的基因产物的表达和聚集的方法以及用于鉴定可用于进行相同的试剂的方法
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Application No.: US13936696Application Date: 2013-07-08
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Publication No.: US09226935B2Publication Date: 2016-01-05
- Inventor: Tzu-Hao Cheng , Chia-Rung Lu , Tzu-Han Wang , Stanley N. Cohen
- Applicant: Tzu-Hao Cheng , Chia-Rung Lu , Tzu-Han Wang , Stanley N. Cohen
- Applicant Address: TW Taipei US CA Stanford
- Assignee: NATIONAL YANG MING UNIVERSITY,THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY
- Current Assignee: NATIONAL YANG MING UNIVERSITY,THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY
- Current Assignee Address: TW Taipei US CA Stanford
- Agency: Muncy, Geissler, Olds & Lowe, P.C.
- Priority: TW099143336A 20101210
- Main IPC: A61P25/00
- IPC: A61P25/00 ; A61K31/713 ; C12N15/113 ; C12Q1/68 ; A61K51/10
Abstract:
This invention provides a method for modulating the expression of a first gene in a cell wherein the first gene is one containing more than 36 CAG trinucleotide repeats and encoding a protein that form polyglutamine-mediated protein aggregation. Suppression of the first gene is achieved by reducing the expression of SPT4 gene or SUPT4H gene. It can also be achieved by inhibiting the formation of a Spt4/Spt5 complex or a Supt4h/Supt5h complex. Also provided is a method for identifying an agent useful for modulating the expression and aggregation of CAG-expanded gene product, or treating a polyglutamine disease such as Huntington's disease.
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