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

===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 2x10<sup>9</sup> 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 vivo''

====I also decided to add in the notes I took in class just for completeness====
*One of the four human gene therapy trials approved and initiated at the same time
*Based on the results of this trial, the others were halted
*Used CF patients who were in remarkably good health
*Did multiple dosing to find effecting concentration

====Results in the human study:====
*Treatment evoked an immune response
*Inflammation accompanied the immune response
*Results are short lived-- at most 6 weeks
*Because of the immune response, will not be able to do multiple dosing
*Very inefficient transfer of gene of interest-- will do nothing to correct the defect
*"Correction of the CF phenotype of the airway epithelium might be achieved with this strategy"
*"To maintain chronic expression, adenovirus vectors will probably have to be administered repeatedly"
**This is not possible with an immune response