Invention Grant
- Patent Title: Methods for identifying agents useful for modulating the expression and aggregation of CAG-expanded gene product in cells
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Application No.: US14957102Application Date: 2015-12-02
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Publication No.: US09637741B2Publication Date: 2017-05-02
- Inventor: Tzu-Hao Cheng , Chia-Rung Liu , Tzu-Han Wang , Stanley N. Cohen
- Applicant: National Yang-Ming University , The Board of Trustees of the Leland Stanford Junior University
- 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: TW99143336A 20101210
- Main IPC: C12Q1/68
- IPC: C12Q1/68 ; C12N15/113 ; A61K31/713 ; G01N33/50 ; C12N5/00 ; 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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