biostudies-literatureUnknown6(5)Ferrua FTelethonThe Lancet. Haematologye239-e253https://www.ebi.ac.uk/biostudies/studies/S-EPMC6494976biostudies-literatureLentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study.PMC6494976TGT16S01Casiraghi MScala SBarzaghi FAssanelli AANaldini LScaramuzza SAiuti ACalbi VAtkinson GValsecchi MGGiannelli SDott CBrigida ICiceri FCulme-Seymour EJBernardo MEBasso-Ricci LSilvani PFossati CAlbertazzi EMigliavacca MVilla AFiori RGattillo SDionisio FPajno RCanarutto DBartoli AGalimberti SCicalese MPBiasco Lvan Rossem KRoncarolo MGFacchini MWolf HMFerrua FAlbert MHSalerio FAfalseLentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study.2019-01-01T00:00:00Z2019 May2026-04-16T11:37:05.823Z2022-07-22T12:01:30.339ZS-EPMC64949763098178310.1016/S2352-3026(19)30021-3