Patent ID: 11896652
Assignee: THE CHILDREN'S HOSPITAL OF PHILADELPHIA
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 21:
22. A method of treating a human subject with hemophilia B comprising:
administering to said human subject a therapeutically effective amount of a recombinant adeno-associated virus (rAAV) vector comprising a vector genome encapsidated by an AAV5 capsid,
wherein said vector genome comprises at least two AAV inverted terminal repeats (ITR), an expression control element conferring liver tissue-specific expression operably linked with a nucleic acid sequence encoding human Factor IX (FIX) protein, and a polyadenylation signal sequence, wherein said nucleic acid sequence encoding human FIX protein is at least 70% identical to SEQ ID NO:10 and encodes the same human FIX protein encoded by SEQ ID NO:10, and
wherein said therapeutically effective amount of said rAAV vector is a dose ranging from 1×1013 to 1×1014 vector genomes per kilogram (vg/kg) human subject body weight.