http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-9325673-A1

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filingDate 1993-06-04-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_0fef5017d8c30852a8dfa90d005f48a3
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publicationDate 1993-12-23-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber WO-9325673-A1
titleOfInvention In vivo gene therapy with intron-free sequence of interest
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.
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