University of Florida Research Foundation; Incorporated
发明人:
Arun Srivastava,George Vladimirovich Aslanidi,Sergei Zolotukhin,Mavis Agbandje-McKenna,Kim M. Van Vliet,Li Zhong,Lakshmanan Govindasamy
申请号：
US14214011
公开号：
US10011640B2
申请日:
2014.03.14
申请国别(地区):
US
年份:
2018
代理人:
摘要：
Disclosed are capsid-modified rAAV expression vectors, as well as infectious virions, compositions, and pharmaceutical formulations containing them. Also provided are methods of preparing and using the disclosed capsid-protein-mutated rAAV constructs in a variety of diagnostic and therapeutic modalities, including, inter alia, as mammalian cell-targeting delivery agents, and as human gene therapy vectors. Also disclosed are large-scale production methods for capsid-modified rAAV expression vectors, viral particles, and infectious virions having improved transduction efficiencies over those of the corresponding, un-modified, rAAV vectors, as well as use of the disclosed compositions in the manufacture of medicaments for a variety of in vitro and/or in vivo applications.
