TY - JOUR T1 - Cystic fibrosis gene therapy: the first trial JF - Archives of Disease in Childhood JO - Arch Dis Child SP - 844 LP - 844 DO - 10.1136/archdischild-2015-309332 VL - 100 IS - 9 A2 - , Y1 - 2015/09/01 UR - http://adc.bmj.com/content/100/9/844.abstract N2 - 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 … ER -