Gene therapy for cystic fibrosis

Details Gene therapy for cystic fibrosis Gene therapy for cystic fibrosis

C - Chemistry, Metallurgy

12

N

C12N 15/86 (2006.01) A61K 31/70 (2006.01) A61K 48/00 (2006.01) C12Q 1/68 (2006.01)

Patent

CA 2289222

The present invention comprises gene therapy for treating cystic fibrosis (CF). Delivery and expression of a single copy of a normal CFTR gene leads to stable correction of the Cl channel regulation defect present in CF epithelial cells. The present invention includes recombinant viral and plasmid vectors, alternative CFTR gene delivery strategies, and transduced CF cells and cell lines carrying a recombinant gene for functional CFTR. CF epithelial complementation through transduction of the present invention also provides an assay for determining the validity of other putative CF mutations.

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