Methods for identifying and compounds useful for increasing the functional activity and cell surface expression of CF-associated mutant cystic fibrosis transmembrane conductance regulator
David Frederik Fischer;Amelia Katie Scaffidi;Michela Tessari;Marjet Roseboom;Richard Antonius Jozef Janssen
发明人:
David Frederik Fischer,Richard Antonius Jozef Janssen,Marjet Roseboom,Amelia Katie Scaffidi,Michela Tessari
申请号：
US13063333
公开号：
US08765376B2
申请日:
2009.09.10
申请国别(地区):
US
年份:
2014
代理人:
摘要：
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).
