Inventors:
- Gainesville FL, US
Li Zhong - Boxborough MA, US
Sergei Zolotukhin - Gainesville FL, US
George Vladimirovich Aslanidi - Austin MN, US
Kim M. Van Vliet - Gainesville FL, US
Chen Ling - Gainesville FL, US
Assignee:
University of Florida Research Foundation, Incorporated - Gainesville FL
International Classification:
C07K 14/005
A61K 48/00
C12N 15/86
C12N 15/864
A61K 35/76
C12N 7/00
Abstract:
Disclosed are next-generation multi-mutated capside protein-modified rAAV expression vector, as well as infectious virions, compositions, and pharmaceutical formulations that include them. Also disclosed are methods of preparing and using these high transduction efficiency vector constructs in a variety of therapeutic applications including, inter alia, as delivery agents for the treatment or amelioration of one or more diseases or abnormal conditions in an affected mammal using in vivo and/or ex situ viral vector-based gene therapy protocols. Also disclosed are large-scale production methods for the multi-mutated, capsid-modified rAAV expression vector, viral particles, and infectious virions, as well as use of the disclosed compositions in the manufacture of medicaments for use in a variety of in vitro and/or in vivo therapeutic methodologies.
