Patent ID: 11939590
Assignee: THE CHILDEN'S HOSPITAL OF PHILADELPHIA
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 0:
1. A method for delivering a heterologous polynucleotide sequence into the liver of a mammal, comprising administering an adeno-associated virus (AAV) vector comprising the heterologous polynucleotide sequence which encodes a blood coagulation factor, wherein the heterologous polynucleotide sequence is operably linked to an expression control element, wherein the expression control element is active in liver, and wherein said AAV vector comprises a VP1 sequence comprising the amino acid sequence set forth as SEQ ID NO:1, to said mammal thereby delivering the heterologous polynucleotide sequence encoding the blood coagulation factor into the liver of the mammal.