BibTex format
@article{Stern:1997,
author = {Stern, M and Phillips, J and Jaffe, A and Farley, R and Chadwick, S and Davies, J and Smith, S and Browning, J and Hodson, ME and Durham, S and Li, D and Jeffery, P and Scallah, M and Balfour, R and Cheng, S and Smith, A and Meeker, D and Geddes, DM and Alton, EWFW},
journal = {Thorax},
title = {A double blind placebo controlled trial of pulmonary and nasal administration of liposome-mediated CFTR gene transfer in CF subjects},
volume = {52},
year = {1997}
}
RIS format (EndNote, RefMan)
TY - JOUR
AB - We and others have previously shown functional changes folllowing liposome-mediated CFTR gene transfer to the nasal epithelium of CF subjects. We have now studied the safety and efficacy of this approach in the lungs and nose of CF subjects in a double blind placebo controlled study. 8 Subjects were randomised to receive DNA liposome complex or lipid alone by nebulisation into the lungs with 8 further subjects receiving the liposome alone. One week later, the same subjects received the same treatment by nasal administration. Safety was assessed clinically, radiographically, by pulmonary function and histological analysis. Efficacy was assessed by analysis of CFTR DNA, mRNA, in vivo potential difference, SPQ fluorescence, bacterial adherence and mucociliary clearance (nose only). The majority of subjects receiving the active complex noted mild flu-like symptoms which resolved within 36 hours. A proportion of both active and placebo subjects noted mild respiratory symptoms over a period of 12 hours following pulmonary administration. No specific treatment was required for either event. Pulmonary administration resulted in a significant (p<0.05) degree of correction of the chloride abnormality in the active, but not placebo treated subjects. No alterations in the sodium transport were detected. A similar pattern was observed following nasal administration. This study provides proof-of-principle that cationic liposome mediated CFTR gene transfer is able to influence significantly the underlying electrophysiological defect in the lungs of cystic fibrosis patients.
AU - Stern,M
AU - Phillips,J
AU - Jaffe,A
AU - Farley,R
AU - Chadwick,S
AU - Davies,J
AU - Smith,S
AU - Browning,J
AU - Hodson,ME
AU - Durham,S
AU - Li,D
AU - Jeffery,P
AU - Scallah,M
AU - Balfour,R
AU - Cheng,S
AU - Smith,A
AU - Meeker,D
AU - Geddes,DM
AU - Alton,EWFW
PY - 1997///
SN - 0040-6376
TI - A double blind placebo controlled trial of pulmonary and nasal administration of liposome-mediated CFTR gene transfer in CF subjects
T2 - Thorax
VL - 52
ER -