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In August, Archivist reported on a successful trial of new molecules that enhance function of cystic fibrosis (CF) transmembrane conductance regulator (CFTR) protein at an intracellular level (ADC doi:10.1136/archdischild-2015-309089). Hot on its heels comes a report of the first Phase 2b trial of another completely novel treatment: gene therapy, which involves transferring correct copies of CFTR DNA into lung epithelial cells, by administering it in a complex with cationic liposomes in nebulised form (Alton …
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