COMPOSITIONS AND METHODS FOR TREATMENT OF CYSTIC FIBROSIS AND DISEASES ASSOCIATED WITH ABERRANT PROTEIN CELLULAR PROCESSING

摘要

The disclosure relates to resorcylic acid lactones and indolinone-containing compounds for use in treatment of diseases associated with aberrant protein processing, such as cystic fibrosis (CF; mucoviscidosis). The disclosure more generally relates to treatment of aberrant protein processing, such as errors in protein folding, trafficking or post-translational modification.