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) C07K 14/47 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2091882

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.

Thérapie génétique pour le traitement de la fibrose kystique (CF). L'apport et l'expression d'une seule copie d'un gène normal de régulation de la conductance transmembrane de fibrose kystique (CFTR) permet d'obtenir une correction stable du défaut de régulation du canal Cl existant dans les cellules épithéliales de CF. La présente invention a trait notamment à des vecteurs de recombinaison de virus et de plasmide, à des stratégies possibles d'apport de gènes CFTR, ainsi qu'à des cellules de CF transmises et à des lignes de cellules véhiculant un gène de recombinaison CFTR fonctionnel. La complémentation épithéliale de la CF au moyen de la transduction de la présente invention est aussi un test permettant de vérifier la validité d'autres mutations putatives de CF.

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