A novel recombinant adeno-associated virus vector packaging system with HSV-1 amplicon providing helper functions.
A novel packaging system for producing recombinant adeno-associated virus (rAAV) vector was described. Instead of the conventional method for rAAV production by two-plasmid co-transfection followed by superinfection with adenovirus 5, an HSV-1 amplicon system expressing AAV-2 rep and cap genes from their native promoters was used to provide complete helper functions for rAAV replicating and packaging. This HSV-1 amplicon stock consisted of two kinds of infectious HSV-1 virions, a replicating-defective HSV-I amplicon pseudovirus harboring multi-copies of AAV-2 rep and cap gene and a temperature-sensitive HSV-1 mutant strain ts-KOS. High-titer rAAV was generated with this new packaging system. This packaging system gives a simple and scaleable process for rAAV production.
Shu
Y
Y
State Key Laboratory for Molecular Virology and Genetic Engineering, 100052, Beijing, China.
Wu
X
X
Yang
T
T
Gong
H
H
Hou
Y
Y
Yan
Z
Z