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：
AU2012340567
公开号：
AU2012340567B2
申请日:
2012.11.21
申请国别(地区):
AU
年份:
2017
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.
