Compositions and Methods for Inhibiting the Interaction Between CFTR and CAL
AssigneeTrustees of Dartmouth College
InventorsDean R. Madden, Patrick R. Cushing, Prisca Boisguearin, Rudolph Volkmer, Lars Vouilleme
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Abstract
The present invention features compositions and methods for increasing the cell surface expression of degradation-prone CFTR proteins and preventing or treating cystic fibrosis. The invention provides peptides and peptidomimetics that selectively inhibit the interaction between CAL and mutant CFTR proteins, thereby stabilizing the CFTR and facilitating transport of the same to the cell surface.
Claims26
A method for increasing cell surface expression of a degradation-prone Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein comprising contacting a cell expressing a degradation-prone CFTR with an effective amount of an agent that selectively inhibits the interaction between the degradation-prone CFTR and CFTR-Associated Ligand thereby increasing cell surface expression of the degradation-prone CFTR protein as compared to cell surface expression in the absence of the agent.
A method for preventing or treating cystic fibrosis comprising administering to a subject in need of treatment an effective amount of an agent that selectively inhibits the interaction between a degradation-prone Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) and CFTR-Associated Ligand thereby preventing or treating the subject's cystic fibrosis.
An agent for inhibiting the interaction between a degradation-prone Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) and CFTR-Associated Ligand (CAL) comprising a peptide having the amino acid sequence of SEQ ID NO:1, or a derivative or peptidomimetic thereof.
Dependencies are inferred from the claim text.