Patent ID: 9629930
Date: 2017-04-25
CPC Classifications: A61K,A61N,A61P,C12N,C12Y

Claim:
1. A method for treating hemophilia B in a subject, the method comprising integrating a sequence encoding a functional Factor IX (FIX) protein or fragment thereof into the genome of a cell using at least one zinc finger nuclease, wherein the zinc finger nuclease comprises an engineered zinc finger protein DNA-binding domain that binds to a target site in an endogenous Factor IX (FIX) gene, wherein the DNA-binding domain comprises four or five zinc finger recognition regions ordered F1 to F4 or F1 to F5 from N-terminus to C-terminus, and wherein (i) when the DNA-binding domain comprises five zinc finger recognition regions, F1 to F5 comprise the following amino acid sequences: wherein the DNA-binding domain binds to the target site shown in SEQ ID NO:3; and (ii) when the DNA-binding domain comprises four zinc finger recognition regions, F1 to F4 comprise the following amino acid sequences: wherein the DNA-binding domain binds to the target site shown in SEQ ID NO:9.