Gene therapy vector for cystic fibrosis
AssigneeThe Regents of the University of Michigan
InventorsFrancis S. Collins, James M. Wilson
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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.
Claims16
A recombinant viral vector for treating a defect in the gene for cystic fibrosis transmembrane regulator in a target cell, the vector comprising:
- a) the DNA of or corresponding to at least a portion of the genome of a virus which portion is capable of infecting the target cells;
- and
- b) a normal cystic fibrosis transmembrane regulator gene operatively linked to the DNA and capable of expression in the target cell in vivo or in vitro.
A CF cell which expresses a normal cystic fibrosis transmembrane regulator gene introduced therein through retroviral transduction.
Dependencies are inferred from the claim text.