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Despite the advances in gene therapy and in oligonucleotide (ON) chemistry, efficient cellular delivery remains an obstacle. Most current transfection reagents suffer from low efficacy or high cytotoxicity. In this report, we describe the synergism between lipid and dendrimer delivery vectors to enhance the transfection efficiency, while avoiding high toxicity. We screened a library of 20 peptide dendrimers representing three different generations and evaluated their capability to deliver a single-stranded splice-switching ON after formulating with lipids (DOTMA/DOPE). The transfection efficiency was analyzed in 5 reporter cell lines, in serum-free and serum conditions, and with 5 different formulation protocols. All formulations displayed low cytotoxicity to the majority of the tested cell lines. The complex sizes were 

Original publication




Journal article


Eur J Pharm Biopharm

Publication Date





29 - 40


Delivery vectors, Gene therapy, Peptide dendrimers, Splice-switching oligonucleotides, Synergism, Transfection, Animals, Cell Line, Dendrimers, Female, Gene Transfer Techniques, Genes, Reporter, Genetic Therapy, Genetic Vectors, HeLa Cells, Humans, Lipids, Mice, Oligonucleotides, Particle Size, Peptides, Tissue Distribution, Transfection