Gene therapy vector for cystic fibrosis

Details Gene therapy vector for cystic fibrosis Gene therapy vector for cystic fibrosis

1990-09-18

1993-08-31

Wax, Robert A.

Chemistry: molecular biology and microbiology

Spore forming or isolating process

4351723, 435 6, 435 691, 435 29, C12P 2106, C12N 500, C12N 1500, C12Q 168

Patent

active

052408469

ABSTRACT:
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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