Zinc finger nuclease for the cftr gene and methods of use thereof
AssigneesThe General Hospital Corporation, University of Iowa Research Foundation
InventorsPaul McCray, Morgan Maeder, Jae Keith Joung
Life of this patent
Estimating the term…
Abstract
The present invention provides new zinc finger proteins and zinc finger nuclease (ZFNs) that find particular using in repairing the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Claims39
A zinc three-finger binding domain that targets a nucleotide sequence selected from the group consisting of GTGGAATTA (SEQ ID NO:1) and GAGTGGTTA (SEQ ID NO:2).
A zinc three-finger binding domain dimer that targets a double-stranded nucleic acid comprising a first monomer that targets nucleotide sequence GTGGAATTA (SEQ ID NO:1) in one strand and a second monomer that targets nucleotide sequence GAGTGGTTA (SEQ ID NO:2) in the other strand.
A vector comprising a nucleic acid segment encoding a zinc three-finger binding domain that targets a nucleotide sequence selected from the group consisting of GTGGAATTA (SEQ ID NO:1) and GAGTGGTTA (SEQ ID NO:2), said nucleic acid under the control of a promoter operable in a eukaryotic cell.
A method of promoting recombination within a CTFR gene in a human cell comprising contacting said cell with a first zinc three-finger binding domain that targets a nucleotide sequence GTGGAATTA (SEQ ID NO:1) and a second zinc three-finger binding domain that targets a nucleotide sequence GAGTGGTTA (SEQ ID NO:2), wherein each of said first and second zinc three-finger binding domains are linked to a non-specific nuclease.
Dependencies are inferred from the claim text.