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Gene therapy for the treatment of cystic fibrosis

Overview of attention for article published in The Application of Clinical Genetics, May 2012
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206 Mendeley
Title
Gene therapy for the treatment of cystic fibrosis
Published in
The Application of Clinical Genetics, May 2012
DOI 10.2147/tacg.s8873
Pubmed ID
Authors

Jane Davies, Burney, Eric Alton

Abstract

Gene therapy is being developed as a novel treatment for cystic fibrosis (CF), a condition that has hitherto been widely-researched yet for which no treatment exists that halts the progression of lung disease. Gene therapy involves the transfer of correct copies of cystic fibrosis transmembrane conductance regulator (CFTR) DNA to the epithelial cells in the airways. The cloning of the CFTR gene in 1989 led to proof-of-principle studies of CFTR gene transfer in vitro and in animal models. The earliest clinical trials in CF patients were conducted in 1993 and used viral and non-viral gene transfer agents in both the nasal and bronchial airway epithelium. To date, studies have focused largely on molecular or bioelectric (chloride secretion) outcome measures, many demonstrating evidence of CFTR expression, but few have attempted to achieve clinical efficacy. As CF is a lifelong disease, turnover of the airway epithelium necessitates repeat administration. To date, this has been difficult to achieve with viral gene transfer agents due to host recognition leading to loss of expression. The UK Cystic Fibrosis Gene Therapy Consortium (Imperial College London, University of Edinburgh and University of Oxford) is currently working on a large and ambitious program to establish the clinical benefits of CF gene therapy. Wave 1, which has reached the clinic, uses a non-viral vector. A single-dose safety trial is nearing completion and a multi-dose clinical trial is shortly due to start; this will be powered for clinically-relevant changes. Wave 2, more futuristically, will look at the potential of lentiviruses, which have long-lasting expression. This review will summarize the current status of translational research in CF gene therapy.

Twitter Demographics

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Mendeley readers

The data shown below were compiled from readership statistics for 206 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
United Kingdom 1 <1%
India 1 <1%
Unknown 204 99%

Demographic breakdown

Readers by professional status Count As %
Student > Bachelor 86 42%
Student > Master 21 10%
Student > Ph. D. Student 20 10%
Student > Doctoral Student 12 6%
Researcher 9 4%
Other 18 9%
Unknown 40 19%
Readers by discipline Count As %
Biochemistry, Genetics and Molecular Biology 60 29%
Medicine and Dentistry 27 13%
Agricultural and Biological Sciences 25 12%
Pharmacology, Toxicology and Pharmaceutical Science 17 8%
Immunology and Microbiology 8 4%
Other 29 14%
Unknown 40 19%