2016
DOI: 10.17925/erpd.2016.02.02.48
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Cystic Fibrosis Gene Therapy – Not Low-hanging Fruit

Abstract: The last 25 years have shown that it has been comparatively slow and difficult to develop cystic fibrosis (CF) gene therapy; the lung is a complex target organ. However, research has steadily progressed and recently it was shown that non-viral gene therapy can stabilise CF lung disease. These data, in addition to the development of potent lentiviral vectors, have renewed interest in CF gene therapy within academia and industry.

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