首页> Molecular therapy : the journal of the American Society of Gene Therapy> A promoterless AAV6.2FF-based lung gene editing platform for the correction of surfactant protein B deficiency.

A promoterless AAV6.2FF-based lung gene editing platform for the correction of surfactant protein B deficiency.

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第一作者： Sylvia P,Thomas

第一单位： Department of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada.

作者： Sylvia P,Thomas ^[1] ; Jakob M,Domm ^[1] ; Jacob P,van Vloten ^[1] ; Liqun,Xu ^[2] ; Arul,Vadivel ^[2] ; Jacob G E,Yates ^[1] ; Yanlong,Pei ^[1] ; Joelle,Ingrao ^[1] ; Laura P,van Lieshout ^[1] ; Sergio R,Jackson ^[3] ; Jessica A,Minott ^[1] ; Adithya,Achuthan ^[2] ; Yeganeh,Mehrani ^[1] ; Thomas M,McAusland ^[1] ; Wei,Zhang ^[4] ; Khalil,Karimi ^[1] ; Andrew E,Vaughan ^[3] ; Jondavid,de Jong ^[1] ; Martin H,Kang ^[5] ; Bernard,Thebaud ^[2] ; Sarah K,Wootton ^[6]

作者单位： Department of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada. ^[1] Regenerative Medicine Program, The Ottawa Hospital Research Institute (OHRI), Ottawa, ON, Canada; Department of Cellular and Molecular Medicine, University of Ottawa, Ottawa, ON, Canada; Neonatology, Department of Pediatrics, Children's Hospital of Eastern Ontario (CHEO), and CHEO Research Institute, Ottawa, ON K1Y 4E9, Canada. ^[2] Department of Biomedical Sciences, University of Pennsylvania School of Veterinary Medicine, Philadelphia, PA 19104, USA. ^[3] Department of Molecular and Cellular Biology, University of Guelph, Guelph, ON, Canada. ^[4] Department of Pediatrics, Darby Children's Research Institute, Medical University of South Carolina, Charleston, SC 29425, USA. ^[5] Department of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada. Electronic address: kwootton@uoguelph.ca. ^[6]

关键词 AAV AAV6.2FF CRISPR-Cas9 HDR adeno-associated virus alveolar type II cells gene editing gene ride homology directed repair intranasal surfactant protein B deficiency surfactant protein C