Method of identifying agents for the treatment of cystic fibrosis caused by the mutation f508del

Abstract

A method of identifying agents for the treatment of Cystic Fibrosis (CF) caused by the mutation F508del in the gene encoding the cystic fibrosis transmembrane conductance regulator protein (F508del-CFTR), wherein a candidate agent contacts with at least one gene sequence selected from SEQ ID NOs: 228 to 478 or with its protein product as therapeutic targets. The ability of the candidate agent to modulate the activity of the F508del-CFTR protein and its plasma membrane traffic is determined. Gene sequences SEQ ID NOs: 228 to 478 were identified by a high-throughput siRNA cell-based microscopy screen aimed at identifying genes which enhance F508del-CFTR plasma membrane levels upon knock-down, as potential candidates for drug therapy in the treatment of CF. Further disclosing formulations comprising agents capable of targeting at least one gene sequence selected from SEQ ID NOs: 228 to 478 or their protein product.

Dependencies are inferred from the claim text.