IMPROVED CONCEPTS FOR THE TREATMENT OF GENETIC DISORDERS WITH HIGH-CAPACITY PLAL-GENERATED GOLD NANOPARTICLES

摘要

The present invention relates to conjugated gold nanoparticles, preferably for the use in the treatment of a monogenetic disorder resulting from a mutation in a gene coding for a liver-specific and/or liver-expressed protein, comprising laser-ablated gold nanoparticles, polyethylenimine (PEI) and/or derivatives and/or salts thereof and a nucleic acid molecule. Furthermore, the present invention relates to the use of such gold nanoparticles, a method for the preparation of conjugated gold nanoparticles, a nanoparticle-based delivery system and the use of such delivery system. In addition, the present invention relates to a method for transfection of target cells, a transfected target cell as well as a vector for the expression of a liver- specific and/or liver-expressed protein.