METHODS FOR IDENTIFYING AND COMPOUNDS USEFUL FOR INCREASING THE FUNCTIONAL ACTIVITY AND CELL SURFACE EXPRESSION OF CF-ASSOCIATED MUTANT CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR

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).