Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (cftr) gene

Abstract

Nucleases and methods of using these nucleases for alteration of a CFTR gene and generation of cells and animal models.

What is claimed is: 
     
         1 . A method of modifying a Surfactant Protein B (SP-B) gene in a cell, the method comprising;
 cleaving the SP-B gene with one or more zinc finger nucleases that bind to a target site in the SP-B gene.   
     
     
         2 . The method of  claim 1 , wherein the modification is selected from the group consisting of an insertion, a deletion, a substitution and combinations thereof. 
     
     
         3 . The method of  claim 1 , further comprising introducing an exogenous sequence into the SP-B gene. 
     
     
         4 . The method of  claim 1 , wherein the modification corrects a mutation in the SP-B gene. 
     
     
         5 . The method of  claim 4 , wherein the mutation is a121 ins2 mutation. 
     
     
         6 . A method of generating a model system for the study of Surfactant Protein B deficiency, the method comprising modifying cells according to the method of  claim 1 . 
     
     
         7 . The method of  claim 6 , wherein the model system comprises a cell line. 
     
     
         8 . The method of  claim 6 , wherein the model system comprises a non-human animal. 
     
     
         9 . A method of treating Surfactant Protein B deficiency in a subject, the method comprising modifying a SP-B gene in one or more cells of the subject according to the method of  claim 4 . 
     
     
         10 . The method of  claim 9 , wherein the cell is modified in vitro and the cell is administered to the subject.