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Taking stock of gene therapy for cystic fibrosis

Authors
Journal
BMJ Open Respiratory Research
1465-9921
Publisher
BMJ
Publication Date
Volume
1
Issue
2
Identifiers
DOI: 10.1186/rr16
Keywords
  • Commentary
Disciplines
  • Medicine

Abstract

The identification of the cystic fibrosis (CF) gene opened the way for gene therapy. In the ten years since then, proof of principle in vitro and then in animal models in vivo has been followed by numerous clinical studies using both viral and non-viral vectors to transfer normal copies of the gene to the lungs and noses of CF patients. A wealth of data have emerged from these studies, reflecting enormous progress and also helping to focus and define key difficulties that remain unresolved. Gene therapy for CF remains the most promising possibility for curative rather than symptomatic therapy.

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