Journal article
Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis
Cell (Cambridge), Vol.75(2), pp.207-216
1993
DOI: 10.1016/0092-8674(93)80063-K
PMID: 7691415
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.
Details
- Title: Subtitle
- Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis
- Creators
- Joseph Zabner - Howard Hughes Medical Institute Departments of Internal Medicine and Physiology and Biophysics, University of Iowa College of Medicine, Iowa City, Iowa 52242, USALarry A Couture - Genzyme Corporation One Mountain Road, Framingham, Massachusetts 01701, USARichard J Gregory - Genzyme Corporation One Mountain Road, Framingham, Massachusetts 01701, USAScott M Graham - Department of Otolaryngology University of Iowa College of Medicine Iowa City, Iowa 52242, USAAlan E Smith - Genzyme Corporation One Mountain Road, Framingham, Massachusetts 01701, USAMichael J Welsh - Howard Hughes Medical Institute Departments of Internal Medicine and Physiology and Biophysics, University of Iowa College of Medicine, Iowa City, Iowa 52242, USA
- Resource Type
- Journal article
- Publication Details
- Cell (Cambridge), Vol.75(2), pp.207-216
- Publisher
- Elsevier Inc
- DOI
- 10.1016/0092-8674(93)80063-K
- PMID
- 7691415
- ISSN
- 0092-8674
- eISSN
- 1097-4172
- Language
- English
- Date published
- 1993
- Academic Unit
- Neurology; Molecular Physiology and Biophysics; Pulmonary, Critical Care, and Occupational Medicine; Neurosurgery; Otolaryngology; Internal Medicine
- Record Identifier
- 9984006407302771
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