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

Claim 17:
18. A method of delivering a heterologous polynucleotide encoding a blood coagulation factor to a mammal deficient in said blood coagulation factor expression or function, comprising: (a) providing an AAV vector comprising the heterologous polynucleotide which encodes a blood coagulation factor that can correct for said deficient blood coagulation factor expression or function, 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; and (b) administering an amount of the AAV vector results in delivering the heterologous polynucleotide to the mammal, wherein said blood coagulation factor is expressed by a liver cell of the mammal.