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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(Introduction)
(Abstract)
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===Abstract===
===Abstract===
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* They administered a recombinant adenovirus vector (AdCFTR) containing the normal human CFTR cDNA into the nasal and bronchial epithelium of 4 individuals with CF.
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* They found the vector expresses the CFTR cDNA in the respiratory epithelium in vivo.
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* 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)
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* 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
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