Episomal expression cassettes for gene therapy

C - Chemistry – Metallurgy – 12 – N

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C12N 15/85 (2006.01) A61K 9/127 (2006.01) A61K 31/70 (2006.01) A61K 48/00 (2006.01) C07K 14/47 (2006.01) C12N 15/12 (2006.01) C12N 15/88 (2006.01)

Patent

CA 2205076

The invention consists of episomal expression cassettes for expression of a transgene in gene therapy. The expression cassettes consist of regulatory elements of the human cytokeratin gene and a transgene. The invention also includes liposomes for transfection of epithelial tissue with the cassettes in treatment of cystic fibrosis, emphyserma, cancers of epithelial origin arising in the lung or other organs.

L'invention consiste en des cassettes pour l'expression épisomale d'un transgène en thérapie génique. Les cassettes comprennent des éléments régulateurs du gène humain de la cytokératine et un transgène. L'invention concerne également des liposomes pour la transfection du tissu épithélial avec les cassettes dans le traitement de la fibrose kystique, de l'emphysème, des cancers d'origine épithéliale affectant les poumons ou d'autres organes.

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