Article
Evaluation of the efficacy and safety of in vitro, adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator cDNA.
Human gene therapy - 1 Jun 1994
Mittereder N, Yei S, Bachurski C, Cuppoletti J, Whitsett J A, Tolstoshev P, Trapnell B C
Abstract excerpt
Cystic fibrosis (CF) is a common, fatal recessive disease caused by mutations of the cystic fibrosis transmembrane conductance regulator (CFTR) gene manifested by abnormalities in the regulation of chloride ion (Cl-) secretion across the apical membrane of epithelial cells throughout the body. Adenovirus-mediated delivery of the normal CFTR cDNA and correction of the CF epithelial cell Cl- secretory phenotype...
Topics
- Adenoviruses, Human
- Cell Line
- Cystic Fibrosis
- DNA, Recombinant
- Epithelial Cells
- Gene Deletion
- Gene Transfer Techniques
- Genetic Therapy
- Genetic Vectors
- Humans
- Membrane Proteins