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Pore-forming small molecules offer a promising way to tackle cystic fibrosis

In cystic fibrosis, ion-transport abnormalities cause problems in many organs. A small molecule that forms cell-membrane pores allowing ion transport shows therapeutic promise in human cells and a model of the disease.
David N. Sheppard is in the School of Physiology, Pharmacology and Neuroscience, University of Bristol, Bristol BS8 1TD, UK.
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Anthony P. Davis is in the School of Chemistry, University of Bristol, Bristol BS8 4LN, UK.
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Nature 567, 315-317 (2019)

doi: 10.1038/d41586-019-00781-y

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Competing Financial Interests

D.N.S. is the recipient of a Vertex Innovation Award funded by Vertex Pharmaceuticals. The awards are administered by Chameleon, a medical communications company. Vertex Innovation Awards are reviewed by a grant-awarding panel, which selects reviewers to assess the applications and makes a final decision based on the comments of peer reviewers. Members of the grant-awarding panel and peer reviewers are academics and/or clinicians independent of Vertex Pharmaceuticals.

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