Gene therapy for treating hemophilia b

Abstract

Compositions and regimens useful in treating hemophilia B are provided. The method involves administering to the human subject via a peripheral vein by infusion of a suspension of replication deficient recombinant adeno-associated virus (rAAV).

1 . A recombinant adeno-associated virus (rAAV) useful as a liver-directed therapeutic for hemophilia B, said rAAV comprising an AAVrh10 capsid and a vector genome packaged therein, said vector genome comprising:
 (a) an AAV 5′ inverted terminal repeat (ITR);   (b) a coding sequence for a human Factor IX (F9) having coagulation function operably linked to regulatory elements which direct expression of the human Factor IX in liver cells, wherein the coding sequence is SEQ ID NO: 2, and wherein the regulatory elements comprise (i) two copies of an alpha-1 microglobulin/bikunin enhancer and (ii) a thyroid hormone binding globulin (TBG) promoter; and   (c) an AAV 3′ ITR.