THE CHILDREN'S HOSPITAL OF PHILADELPHIA;THE CHILDRENS HOSPITAL OF PHILADELPHIA
发明人:
MINGOZZI, FEDERICO,ANGUELA, XAVIER,WRIGHT, J. FRASER,HIGH, KATHERINE A.
申请号：
CA2856137
公开号：
CA2856137A1
申请日:
2012.11.21
申请国别(地区):
CA
年份:
2013
代理人:
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.
