US 11,939,590 B1
AAV vector compositions and methods for gene transfer to cells, organs and tissues
Katherine A. High, Merion Station, PA (US); Federico Mingozzi, Philadelphia, PA (US); Junwei Sun, Philadelphia, PA (US); and Philip Johnson, Wynnewood, PA (US)
Assigned to THE CHILDEN'S HOSPITAL OF PHILADELPHIA, Philadelphia, PA (US)
Filed by The Children's Hospital of Philadelphia, Philadelphia, PA (US)
Filed on Jul. 25, 2018, as Appl. No. 16/045,619.
Application 16/045,619 is a continuation of application No. 14/378,886, abandoned, previously published as PCT/US2013/026695, filed on Feb. 19, 2013.
Claims priority of provisional application 61/600,415, filed on Feb. 17, 2012.
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 48/00 (2006.01); A61K 38/36 (2006.01); C12N 15/12 (2006.01); C12N 15/86 (2006.01); C12N 15/864 (2006.01)
CPC C12N 15/86 (2013.01) [C12N 2750/14143 (2013.01)] 19 Claims
 
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.