Invention Grant
US08765376B2 Methods for identifying and compounds useful for increasing the functional activity and cell surface expression of CF-associated mutant cystic fibrosis transmembrane conductance regulator
失效
用于鉴定和用于增加CF相关突变型囊性纤维化跨膜传导调节物的功能活性和细胞表面表达的化合物的方法
- Patent Title: Methods for identifying and compounds useful for increasing the functional activity and cell surface expression of CF-associated mutant cystic fibrosis transmembrane conductance regulator
- Patent Title (中): 用于鉴定和用于增加CF相关突变型囊性纤维化跨膜传导调节物的功能活性和细胞表面表达的化合物的方法
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Application No.: US13063333Application Date: 2009-09-10
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Publication No.: US08765376B2Publication Date: 2014-07-01
- Inventor: David Frederik Fischer , Richard Antonius Jozef Janssen , Marjet Roseboom , Amelia Katie Scaffidi , Michela Tessari
- Applicant: David Frederik Fischer , Richard Antonius Jozef Janssen , Marjet Roseboom , Amelia Katie Scaffidi , Michela Tessari
- Applicant Address: BE Mechelen
- Assignee: Galapagos NV
- Current Assignee: Galapagos NV
- Current Assignee Address: BE Mechelen
- Agency: Klauber & Jackson, LLC
- International Application: PCT/EP2009/061732 WO 20090910
- International Announcement: WO2010/029118 WO 20100318
- Main IPC: A61K31/70
- IPC: A61K31/70 ; A61K31/7088 ; A61P1/16 ; C12N15/11 ; G01N33/68 ; C07H21/02 ; C12Q1/68 ; C07H21/04
Abstract:
The present invention relates to agents, and methods for identifying compounds, which agents and compounds result in the modulation of cellular trafficking of proteins in particular that of CF-associated mutant Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). In addition, the invention relates to compositions and methods for the use thereof in treating conditions that are characterized by an ER-associated protein misfolding and abnormal cellular trafficking of disease-associated proteins, including cystic fibrosis (CF).
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