CFTR with a partially deleted R domain and uses thereof

Abstract

The present invention offers new therapies for treating Cystic Fibrosis (CF), that are based on novel DNA molecules and proteins encoded by the DNA molecules. The present invention features DNA molecules encoding CFTR having a partially deleted R domain. The partial deletions in the R domain are between residues 708 and 835 of the wild-type CFTR.

1 . A CFTR protein having a deletion in the R domain selected from the group consisting of SEQ ID NO:10. 
     
     
         2 . A CFTR protein having a deletion in the R domain, wherein said CFTR protein provides low constitutive chloride ion current in CFTR channels, and wherein said CFTR provides a functional chloride ion channel in CF airway epithelia cells, and comprising a CFTR protein with a deletion of amino acids 708-759. 
     
     
         3 . The CFTR protein of  claim 2 , wherein said CFTR protein is selected from the group consisting of SEQ ID NO:10. 
     
     
         4 . A CFTR protein comprising approximately 95% homology to the protein of SEQ ID NO:10 wherein said CFTR protein has bases 708-759 deleted from wild-type CFTR sequence.