FIELD: medicine.
SUBSTANCE: invention relates to field of biotechnology, genetic engineering and virology. In addition to SIN-vector system contains besides first helper plasmid, intended for expression of viral fusion protein gag-pol, and second helper plasmid, intended for expression of envelope protein (env) of retrovirus. First and second helper plasmids, contained in packaging cell line or applied for its short-term transfection, serve for creation of non-replicating (RCR-incompetent) viral particles. Viral particles contain RNA, which contains on 3'-end SIN-LTR, claimed in invention, where RNA can have therapeutically effective segment, which, for instance, represents transgene. For this purpose extensive deletion of U3-region, which in the process of reverse transcription is copied in 5' LTR, is provided in 3'-SIN-LTR. In addition, all coding ASLV regions, as well as retroviral splicing donor sites are removed from SIN-vector. Invention can be applied in genetic therapy.
EFFECT: viral self-inactivating (SIN) vector, based on virus of sarcoma and avian leukaemia virus (ASLV), and cleaving-packaging system are described.
24 cl, 15 dwg, 2 tbl, 4 ex
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Authors
Dates
2015-10-27—Published
2010-05-17—Filed