Gene therapy vector for cystic fibrosis
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United States of America Patent
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Aug 31, 1993
Issued Date -
N/A
app pub date -
Sep 18, 1990
filing date -
Aug 22, 1989
priority date (Note) -
Expired
status (Latency Note)
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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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