Novel transgene expression system for increased persistence

Details Novel transgene expression system for increased persistence Novel transgene expression system for increased persistence

C - Chemistry, Metallurgy

12

N

C12N 15/86 (2006.01) A61K 48/00 (2006.01) C07K 14/47 (2006.01) C12N 15/861 (2006.01)

Patent

CA 2287076

The invention is directed to a transgene expression system comprising a transcription unit which contains a transgene operably linked to expression control sequences, preferably the CMV promoter, and which is delivered simultaneously with all or part of the adenovirus E4 genomic region to a cell in order to facilitate persistent expression of the transgene. The components of the transgene expression system can be delivered by vectors including plasmids and/or viruses and may be complexed with cationic amphiphiles to facilitate entry into a cell. The invention is also directed to methods for the production of the transgene expression system. The invention is further directed to compositions that contain the transgene expression system and to methods for the use of such compositions to deliver transgenes encoding biologically active proteins to cells.

La présente invention concerne un système d'expression de transgène comprenant une unité de transcription qui comprend un transgène fonctionnellement lié à des séquences de régulation d'expression, de préférence, le promoteur CMV, et qui est administrée à une cellule, simulténément, avec tout ou partie de la région génomique E4 d'adénovirus, de façon à faciliter une expression persistante du transgène. Les composants du système d'expression de transgène peuvent être administrés par des vecteurs comprenant des plasmides et/ou des virus et peuvent former des complexes avec des amphiphiles cationiques de façon à faciliter l'introduction dans une cellule. La présente invention concerne également des procédés de production du système d'expression de transgène. La présente invention concerne, en outre, des compositions contenant le système d'expression de transgène et des procédés d'utilisation de ces compositions de façon à administrer à des cellules des transgènes codant biologiquement pour des protéines biologiquement actives.

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