| Title |
Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis
|
|---|---|
| Published in |
Cell, October 1993
|
| DOI | 10.1016/0092-8674(93)80063-k |
| Pubmed ID | |
| Authors | |
| Abstract |
To evaluate the potential of direct transfer of cystic fibrosis transmembrane conductance regulator (CFTR) cDNA for the treatment of cystic fibrosis (CF), we administered an E1-deficient adenovirus, encoding CFTR, to a defined area of nasal airway epithelium of three individuals with CF. This treatment corrected the Cl- transport defect that is characteristic of CF-affected epithelia. After treatment, there was a decrease in the elevated basal transepithelial voltage, and the normal response to a cAMP agonist was restored. We found no evidence of viral replication or virus-associated adverse effects, even at the highest dose tested (25 MOI). These data represent a small step in achieving long-term improvement of CF lung function by gene therapy. |
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Mendeley demographics
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Portugal | 1 | <1% |
| Greece | 1 | <1% |
| Switzerland | 1 | <1% |
| Canada | 1 | <1% |
| Argentina | 1 | <1% |
| Unknown | 182 | 97% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Student > Bachelor | 36 | 19% |
| Student > Ph. D. Student | 33 | 18% |
| Student > Master | 18 | 10% |
| Researcher | 18 | 10% |
| Professor | 10 | 5% |
| Other | 27 | 14% |
| Unknown | 45 | 24% |
| Readers by discipline | Count | As % |
|---|---|---|
| Biochemistry, Genetics and Molecular Biology | 42 | 22% |
| Agricultural and Biological Sciences | 32 | 17% |
| Medicine and Dentistry | 30 | 16% |
| Pharmacology, Toxicology and Pharmaceutical Science | 8 | 4% |
| Immunology and Microbiology | 7 | 4% |
| Other | 17 | 9% |
| Unknown | 51 | 27% |