Gene therapy for cystic fibrosis transmembrane conductance...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/88 (2006.01) A61K 9/127 (2006.01) A61K 9/133 (2006.01) A61K 9/72 (2006.01) A61K 31/70 (2006.01) A61K 47/48 (2006.01) A61K 48/00 (2006.01) C07K 14/00 (2006.01) C07K 14/47 (2006.01) C07K 14/55 (2006.01) C12N 15/12 (2006.01) A61K 39/00 (2006.01)

Patent

CA 2126101

2126101 9312240 PCTABS00023 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 alone are prepared then delivered via aerosol or systemically to the lung alone 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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