Mammalian transformation complex comprising a lipid carrier and DNA encoding CFTR
First Claim
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1. A pharmaceutical composition, said composition comprising a mammalian transcription complex in a pharmaceutically acceptable carrier or diluent;
- said complex comprising a cationic lipid carrier and a plasmid having a transcription or expression cassette comprising a nucleotide sequence encoding a wild-type cystic fibrosis transmembrane conductance regulator protein, wherein said cassette is capable of being expressed to produce said protein in mammalian cells transfected with said cassette; and
wherein said lipid carrier comprises cationic and noncationic lipids, and wherein said complex has a ratio of between about 4;
1 to 1;
10 micrograms DNA to nanomoles cationic lipid; and
said complex does not aggregate in vitro.
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Abstract
Methods and compositions for producing a mammal capable of expressing an exogenously supplied gene in cells of the airway are disclosed. Lipid carrier-nucleic acid complexes or nucleic acid abre are prepared then delivered via aerosol or systemically to the lung abre or lung plus extrapulmonary tissues. The invention provides a direct method for transforming pulmonary cells as a means for treating the manifestations of CF in the lung and involved extrapulmonary tissues.
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Citations
26 Claims
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1. A pharmaceutical composition, said composition comprising a mammalian transcription complex in a pharmaceutically acceptable carrier or diluent;
- said complex comprising a cationic lipid carrier and a plasmid having a transcription or expression cassette comprising a nucleotide sequence encoding a wild-type cystic fibrosis transmembrane conductance regulator protein, wherein said cassette is capable of being expressed to produce said protein in mammalian cells transfected with said cassette; and
wherein said lipid carrier comprises cationic and noncationic lipids, and wherein said complex has a ratio of between about 4;
1 to 1;
10 micrograms DNA to nanomoles cationic lipid; and
said complex does not aggregate in vitro. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24)
- said complex comprising a cationic lipid carrier and a plasmid having a transcription or expression cassette comprising a nucleotide sequence encoding a wild-type cystic fibrosis transmembrane conductance regulator protein, wherein said cassette is capable of being expressed to produce said protein in mammalian cells transfected with said cassette; and
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25. A lit, said kit comprising:
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a first container containing a plasmid having a transcription or expression cassette comprising a nucleotide sequence encoding a wild-type cystic fibrosis transmembrane conductance regulator protein and wherein said cassette is capable of being expressed to produce said protein in mammalian cells transfected with said cassette; and
a second container containing a specific amount of a cationic lipid carrier comprising cationic and noncationic lipids. - View Dependent Claims (26)
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Specification