abstract |
Methods and compositions are provided for introduction of a transgene into a plurality of mammalian tissues in vivo. The method includes the step of incorporating a transfection cassette comprising a nucleotide sequence of interest into a largely non-integrating plasmid and introducing the plasmid into a mammalian host, other than by directly introducing it into a specific tissue. The transfection cassette comprises as operably joined components, a transcriptional and initiation regulatory region, a nucleic acid sequence of interest, and a transcriptional termination regulatory region, wherein said regulatory regions are functional in the cells of a mammalian host, and wherein said nucleic acid sequence of interest is free of introns. Optionally, the transcriptional cassette is complexed with a cationic lipid carrier. The method finds use to modulate the phenotype of mammalian cells in the palliation or treatment of any of a variety of genetic-based disorders. |