Outline 
1. Introduction 
1.1 General background regarding cystic fibrosis 
1.2 Pulmonary manifestations of cystic fibrosis 
1.3 Current therapy for the respiratory manifestations of cystic 
fibrosis 
1.4 New therapies being evaluated for the respiratory manifestations of 
cystic fibrosis 
1.5 Rationale for gene therapy to treat the respiratory manifestations 
of cystic fibrosis 
1.6 Cystic fibrosis transmembrane conductance regulator (CFTR) gene and 
mutations 
1.7 CFTR protein and its function 
1.8 Molecular pathogenesis of cystic fibrosis 
1.9 Cell targets for gene transfer in cystic fibrosis 
1.9.1 General airway morphology 
1.9.2 Normal airway epithelium 
1.9.3 Normal epithelial cytokinetics 
1.9.4 Epithelial lining fluid, pericellular fluid and mucus 
1.9.5 Airway epithelium in CF 
1.10 CFTR gene expression in the airway epithelium 
1.11 Pathogenesis of the respiratory manifestations of cystic fibrosis 
1.11.1 CFTR mutations and respiratory disease 
1.11.2 How abnormal CFTR gene expression causes the characteristic 
airway disease 
1.12 Rationale for using a replication deficient adenovirus containing 
the human CFTR cDNA to treat the respiratory manifestations of 
cystic fibrosis 
2. Replication deficient recombinant adenovirus containing the human CFTR cDNA 
2.1 Overview of the adenovirus 
2.2 Structure of the adenovirus 
2.2.1 Structural proteins 
2.3 Structure of the replication deficient, recombinant adenovirus 
vectors 
2.4 Fabrication and production of AdCFTR 
2.5 Formulation and quality control parameters 
3. Experimental evidence supporting the use of a replication deficient 
recombinant adenovirus containing the human CFTR cDNA to treat the 
respiratory manifestations of cystic fibrosis 
3.1 In vitro evidence 
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Recombinant DNA Research, Volume 16 
