Crystal's "Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis" 1994
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===Abstract=== | ===Abstract=== | ||
- | * | + | * They administered a recombinant adenovirus vector (AdCFTR) containing the normal human CFTR cDNA into the nasal and bronchial epithelium of 4 individuals with CF. |
- | * | + | * They found the vector expresses the CFTR cDNA in the respiratory epithelium in vivo. |
- | * | + | * At 2x109 pfu there was no recombination, complementation, shedding of the vector, or rise in antibody titres. Although, there was a transient and systemic pulmonary syndrome observed (possibly mediated by IL-6) |
- | * | + | * They saw no long term effects |
===Introduction=== | ===Introduction=== |
Revision as of 18:39, 3 March 2010
Abstract
- They administered a recombinant adenovirus vector (AdCFTR) containing the normal human CFTR cDNA into the nasal and bronchial epithelium of 4 individuals with CF.
- They found the vector expresses the CFTR cDNA in the respiratory epithelium in vivo.
- At 2x109 pfu there was no recombination, complementation, shedding of the vector, or rise in antibody titres. Although, there was a transient and systemic pulmonary syndrome observed (possibly mediated by IL-6)
- They saw no long term effects
Introduction
- CF is a common lethal hereditary disorder caused by a mutation on CFTR on chromosome 7
- The disorder is characterized by airway and gastrointestinal disease, the lung manifestations dominate
- The pathogenesis is clearly linked to the lack of CFTR in the respiratory epithelia
- Symptoms in first decade:
- Thick mucus, colonization with infectious bacteria, and chronic airway inflammation
- One approach to prevent respiratory manifestations of CFTR is gene therapy (talked about in abstract)
- Gene therapy must be carried out in vivo, cannot be done ex vitro