Transposons And Rna / Dna Chimeric Oligonucleotides Of The Targeted Gene Therapy

Gene therapy is a new kind of clinical practice which can be defined as simple as the transfer of new genetic material to the cells of an individual with resulting therapeutic benefit to the individual. The main obstacle of clinical application of gene therapy is the limitation of the gene transfer system. The widely used vectors, including viral vectors and nonviral vectors can not meet the demands of clinical trials because of their low efficiency and the absence of targeting. Generally speaking, targeted gene therapy includes cell types targeting, transcriptional targeting and targeted integration of the gene therapy vectors. Now targeted gene therapy is one of the hottest research fields of gene therapy.Transposons are a kind of elements which can jump within a genome. The transposons of Tc1/mariner superfamily are widespread in nematodes, arthropods, insects, fishes and humans. They transpose via a cut-and-paste mechanism. The transposase shares a DD(35)E domain which exists in integrases of retrovirus and bacterial transposase. No host factor is needed in Tc1 transpositon. Recent evidence indicates that the integration of retrovirus and the transposition of Tc1/mariner transposons occur by similar DNA breakage and joining reactions. Since retrovirus and Tc1 transposon share common mechanism for its integration and transposition and the transposase is the only trans factor needed in transposition, a novel gene delivery vector might be developed with Tc1/mariner transposons carried by a vehicle which can bring them into cells.To investigate whether the Tc1 transposon functions in mammalian cells, A transposition-deficiency transposon carrying a NEO gene and the helper plasmid which can express...