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 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

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