VIRUS VECTORS FOR HIGHLY EFFICIENT TRANSGENE DELIVERY
摘要
The invention provides viral vector formulations and methods of uses thereof for delivery of transgenes or therapeutic nucleic acids to human subjects. The formulations include a vector and suitable amounts of empty capsids, viral genome-containing capsids, or viral capsid proteins which are optionally chemically or structurally modified and which bind to neutralizing anti-AAV antibodies thereby reducing or preventing antibody-mediated clearance of the vector, but still allowing the genome-containing (therapeutic) vector to transduce target cells and achieve therapeutic gene transfer.
申请公布号
EP2782596(A4)
申请公布日期
2015.07.29
申请号
EP20120851878
申请日期
2012.11.21
申请人
THE CHILDREN'S HOSPITAL OF PHILADELPHIA
发明人
MINGOZZI, FEDERICO;ANGUELA, XAVIER;WRIGHT, J., FRASER;HIGH, KATHERINE, A.