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rdf:type |
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lifeskim:mentions |
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pubmed:issue |
3
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pubmed:dateCreated |
1997-6-26
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pubmed:abstractText |
Cystic fibrosis (CF) is a common, serious, inherited disease. The major cause of mortality in CF is lung disease, due to the failure of airway epithelial cells to express a functional product of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. A potential treatment for CF lung disease is the expression of CFTR in the airways following gene transfer. We have undertaken a double-blinded, placebo-controlled, clinical study of the transfer of the CFTR cDNA to the nasal epithelium of 12 CF patients. Cationic liposomes complexed with plasmid containing the human CFTR cDNA were administered to eight patients, whilst four patients received placebo. Biopsies of the nasal epithelium taken 7 days after dosing were normal. No significant changes in clinical parameters were observed. Functional expression of CFTR assessed by in vivo nasal potential difference measurements showed transient correction of the CF chloride transport abnormality in two patients (15 days after dosing in one patient). Fluorescence microscopy demonstrated CFTR function ex vivo. In cells from nasal brushings. In total, evidence of functional CFTR gene transfer was obtained in six out of the eight treated patients. These results provide proof of concept for liposome-mediated CF gene transfer.
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pubmed:grant |
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pubmed:language |
eng
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pubmed:journal |
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pubmed:citationSubset |
IM
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pubmed:chemical |
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pubmed:status |
MEDLINE
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pubmed:month |
Mar
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pubmed:issn |
0969-7128
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pubmed:author |
pubmed-author:BiltonDD,
pubmed-author:ColledgeW HWH,
pubmed-author:CuthbertA WAW,
pubmed-author:EvansM JMJ,
pubmed-author:GillD RDR,
pubmed-author:HigginsC FCF,
pubmed-author:HuangLL,
pubmed-author:HydeS CSC,
pubmed-author:LinnL SLS,
pubmed-author:LittlewoodJ MJM,
pubmed-author:MacVinishL JLJ,
pubmed-author:MiddletonP GPG,
pubmed-author:MoffordK AKA,
pubmed-author:RatcliffRR,
pubmed-author:SeddonTT,
pubmed-author:SorgeJJ,
pubmed-author:SouthernK WKW,
pubmed-author:ThomsonAA,
pubmed-author:WebbA KAK
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pubmed:issnType |
Print
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pubmed:volume |
4
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pubmed:owner |
NLM
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pubmed:authorsComplete |
Y
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pubmed:pagination |
199-209
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pubmed:dateRevised |
2009-9-29
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pubmed:meshHeading |
pubmed-meshheading:9135733-Adolescent,
pubmed-meshheading:9135733-Adult,
pubmed-meshheading:9135733-Chlorides,
pubmed-meshheading:9135733-Cystic Fibrosis,
pubmed-meshheading:9135733-Cystic Fibrosis Transmembrane Conductance Regulator,
pubmed-meshheading:9135733-DNA, Complementary,
pubmed-meshheading:9135733-Double-Blind Method,
pubmed-meshheading:9135733-Electrophysiology,
pubmed-meshheading:9135733-Epithelium,
pubmed-meshheading:9135733-Female,
pubmed-meshheading:9135733-Gene Transfer Techniques,
pubmed-meshheading:9135733-Humans,
pubmed-meshheading:9135733-Ion Transport,
pubmed-meshheading:9135733-Liposomes,
pubmed-meshheading:9135733-Male,
pubmed-meshheading:9135733-Microscopy, Fluorescence,
pubmed-meshheading:9135733-Nasal Mucosa
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pubmed:year |
1997
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pubmed:articleTitle |
A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis.
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pubmed:affiliation |
Nuffield Department of Clinical Biochemistry, University of Oxford, John Radcliffe Hospital, UK.
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pubmed:publicationType |
Journal Article,
Clinical Trial,
Randomized Controlled Trial,
Research Support, Non-U.S. Gov't
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