Crystal's "Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis" 1994

From Biol557

Abstract

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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