NANOPARTICLE SYSTEM FOR TRANSFERRING ADJUSTABLE EXPRESSION GENES TO DOPAMINERGIC NEURONS IN LOCUS NIGER.

摘要

Described is a transgene control expression system dependent on doxycycline in a single bifunctional plasmid applied in genic therapy. The invention is intended to adjust, in a specific manner, the transgenic expression in dopaminergic neurons in vivo with potential application in genic therapy of diseases, for instance the Parkinson disease therapy. The invention solves the restriction of the long term and sustained use of neurotrophic factors as therapeutic agents, which are known to produce undesired effects. In addition, the invention solves the restriction of the large molecular size of the adjustable expression systems, which depends on 2 plasmids.