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Targeted Activation of Cystic Fibrosis Transmembrane Conductance Regulator.

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第一作者： Olga,Villamizar

第一单位： Center for Gene Therapy, City of Hope-Beckman Research Institute at the City of Hope, Duarte, CA 91010, USA.

作者： Olga,Villamizar ^[1] ; Shafagh A,Waters ^[2] ; Tristan,Scott ^[1] ; Sheena,Saayman ^[3] ; Nicole,Grepo ^[1] ; Ryan,Urak ^[1] ; Alicia,Davis ^[1] ; Adam,Jaffe ^[4] ; Kevin V,Morris ^[5]

作者单位： Center for Gene Therapy, City of Hope-Beckman Research Institute at the City of Hope, Duarte, CA 91010, USA. ^[1] Faculty of Medicine, School of Women's & Children's Health, University of New South Wales (UNSW), Sydney, NSW, Australia; Molecular and Integrative Cystic Fibrosis Research Centre (miCF_RC), School of Women's & Children's Health, Faculty of Medicine, University of New South Wales, Sydney, NSW, Australia. ^[2] Department of Molecular and Experimental Medicine, The Scripps Research Institute, La Jolla, CA, USA. ^[3] Faculty of Medicine, School of Women's & Children's Health, University of New South Wales (UNSW), Sydney, NSW, Australia; Molecular and Integrative Cystic Fibrosis Research Centre (miCF_RC), School of Women's & Children's Health, Faculty of Medicine, University of New South Wales, Sydney, NSW, Australia; Department of Respiratory Medicine, Sydney Children's Hospital, Sydney, NSW, Australia. ^[4] Center for Gene Therapy, City of Hope-Beckman Research Institute at the City of Hope, Duarte, CA 91010, USA. Electronic address: kmorris@coh.org. ^[5]

关键词 ASO delivery CFTR Tat-CPP cystic fibrosis exosome lncRNA BGas transcriptional regulation