HYDRAZIDE-CONTAINING CFTR INHIBITOR COMPOUNDS AND USES THEREOF

摘要

The invention provides compositions, pharmaceutical preparations and methods for inhibition of cystic fibrosis transmembrane conductance regulator protei n (CFTR) that are useful for the study and treatment of CFTR-mediated diseases and conditions. The compositions and pharmaceutical preparations of the invention may comprise one or more hydrazide-containing compounds, and may additionally comprise one or more pharmaceutically acceptable carriers, excipients and/or adjuvants. The methods of the invention comprise, in certa in embodiments, administering to a patient suffering from a CFTR-mediated disea se or condition, an efficacious amount of a hydrazide-containing compound. In other embodiments the invention provides methods of inhibiting CFTR that comprise contacting cells in a subject with an effective amount of a hydrazi de- containing compound. In addition, the invention features a non-human animal model of CFTR-mediated disease which model is produced by administration of a hydrazide-containing compound to a non-human animal in an amount sufficient to inhibit CFTR.