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Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.
Paulk, Nicole K; Pekrun, Katja; Zhu, Erhua; Nygaard, Sean; Li, Bin; Xu, Jianpeng; Chu, Kirk; Leborgne, Christian; Dane, Allison P; Haft, Annelise; Zhang, Yue; Zhang, Feijie; Morton, Chris; Valentine, Marcus B; Davidoff, Andrew M; Nathwani, Amit C; Mingozzi, Federico; Grompe, Markus; Alexander, Ian E; Lisowski, Leszek; Kay, Mark A.
Affiliation
  • Paulk NK; Departments of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA.
  • Pekrun K; Departments of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA.
  • Zhu E; Translational Vectorology Group, Children's Medical Research Institute, University of Sydney, Sydney, NSW, Australia.
  • Nygaard S; Oregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA.
  • Li B; Oregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA.
  • Xu J; Departments of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA.
  • Chu K; Departments of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA.
  • Leborgne C; Genethon and INSERM U951, Evry, France.
  • Dane AP; Department of Haematology, UCL Cancer Institute, London, UK.
  • Haft A; Oregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA.
  • Zhang Y; Departments of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA.
  • Zhang F; Departments of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA.
  • Morton C; Department of Surgery, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
  • Valentine MB; Cytogenetic Shared Resource, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
  • Davidoff AM; Department of Surgery, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
  • Nathwani AC; Department of Haematology, UCL Cancer Institute, London, UK; Department of Haematology and Katharine Dormandy Haemophilia Centre & Thrombosis Unit, Royal Free London NHS Foundation Trust Hospital, London, UK; National Health Services Blood and Transplant, Watford, UK.
  • Mingozzi F; Genethon and INSERM U951, Evry, France; University Pierre and Marie Curie, Paris, France.
  • Grompe M; Oregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA.
  • Alexander IE; Translational Vectorology Group, Children's Medical Research Institute, University of Sydney, Sydney, NSW, Australia.
  • Lisowski L; Translational Vectorology Group, Children's Medical Research Institute, University of Sydney, Sydney, NSW, Australia; Military Institute of Hygiene and Epidemiology (MIHE), Pulawy, Poland.
  • Kay MA; Departments of Pediatrics and Genetics, Stanford University, Stanford, CA 94305, USA. Electronic address: markay@stanford.edu.
Mol Ther ; 26(1): 289-303, 2018 01 03.
Article in En | MEDLINE | ID: mdl-29055620
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Transduction, Genetic / Genetic Engineering / Dependovirus / Capsid Proteins / Genetic Vectors / Liver Limits: Animals / Female / Humans / Male Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2018 Document type: Article Affiliation country: Estados Unidos Country of publication: Estados Unidos
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Transduction, Genetic / Genetic Engineering / Dependovirus / Capsid Proteins / Genetic Vectors / Liver Limits: Animals / Female / Humans / Male Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2018 Document type: Article Affiliation country: Estados Unidos Country of publication: Estados Unidos