Schambach, Axel,Baum, Christopher,Surth, Julia Debora
申请号：
AU2010247368
公开号：
AU2010247368A1
申请日:
2010.05.17
申请国别(地区):
AU
年份:
2012
代理人:
摘要：
The application provides a viral self-inactivating (SIN) vector based on the avian sarcoma leuosis virus (ASLV) and a split-packaging system which comprises, besides the SIN vector, a first helper plasmid for the expression of the viral fusion protein gag-pol and a second helper plasmid for the expression of the retroviral envelope protein (env). The first and the second helper plasmid, present for example in a packaging cell line or transiently transfected, serve for generating nonreplicating (RCR-incompetent) viral particles which comprise RNA with an SIN-LTR according to the invention at the 3 terminus, where the RNA may have a therapeutically active segment which is referred to for example as a transgene. This 3 SIN LTR comprises an extensive deletion of the U3 region, which is copied into the 5 LTR during reverse transcription. Furthermore, all coding regions of ASLV and the retroviral splice donor site are removed in the SIN vector.
