Recovery of physiological functions using small molecule mimics of missing proteins

摘要

A disease or condition characterized by reduced expression or reduced function of an ion channel comprising administering to a subject in need thereof a therapeutically effective amount of a pore-forming polyene macrolide or a pore-forming derivative thereof. A method for treating is disclosed. For example, the pore-forming polyene macrolide can be amphotericin B (AmB), nystatin, or natamycin. The method can be used to treat cystic fibrosis.