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Correction of a urea cycle defect after ex vivo gene editing of human hepatocytes.
Zabulica, Mihaela; Srinivasan, Raghuraman C; Akcakaya, Pinar; Allegri, Gabriella; Bestas, Burcu; Firth, Mike; Hammarstedt, Christina; Jakobsson, Tomas; Jakobsson, Towe; Ellis, Ewa; Jorns, Carl; Makris, Georgios; Scherer, Tanja; Rimann, Nicole; van Zuydam, Natalie R; Gramignoli, Roberto; Forslöw, Anna; Engberg, Susanna; Maresca, Marcello; Rooyackers, Olav; Thöny, Beat; Häberle, Johannes; Rosen, Barry; Strom, Stephen C.
Afiliação
  • Zabulica M; Department of Laboratory Medicine, Karolinska Institutet, 141 52 Huddinge, Sweden.
  • Srinivasan RC; Department of Laboratory Medicine, Karolinska Institutet, 141 52 Huddinge, Sweden.
  • Akcakaya P; Discovery Sciences, BioPharmaceuticals R&D Unit, AstraZeneca, Gothenburg, Sweden.
  • Allegri G; Division of Metabolism and Children's Research Center, University Children's Hospital, Zürich, Switzerland.
  • Bestas B; Discovery Sciences, BioPharmaceuticals R&D Unit, AstraZeneca, Gothenburg, Sweden.
  • Firth M; Discovery Sciences, BioPharmaceuticals R&D Unit, AstraZeneca, Cambridge, UK.
  • Hammarstedt C; Department of Laboratory Medicine, Karolinska Institutet, 141 52 Huddinge, Sweden.
  • Jakobsson T; Department of Laboratory Medicine, Karolinska Institutet, 141 52 Huddinge, Sweden.
  • Jakobsson T; Department of Clinical Sciences Intervention and Technology, Karolinska Institutet, Stockholm, Sweden.
  • Ellis E; Department of Clinical Sciences Intervention and Technology, Karolinska Institutet, Stockholm, Sweden.
  • Jorns C; Department of Clinical Sciences Intervention and Technology, Karolinska Institutet, Stockholm, Sweden.
  • Makris G; Division of Metabolism and Children's Research Center, University Children's Hospital, Zürich, Switzerland.
  • Scherer T; Division of Metabolism and Children's Research Center, University Children's Hospital, Zürich, Switzerland.
  • Rimann N; Division of Metabolism and Children's Research Center, University Children's Hospital, Zürich, Switzerland.
  • van Zuydam NR; Department of Quantitative Biology, Discovery Sciences, R&D BioPharmaceuticals, AstraZeneca, Gothenburg, Sweden.
  • Gramignoli R; Department of Laboratory Medicine, Karolinska Institutet, 141 52 Huddinge, Sweden.
  • Forslöw A; Discovery Sciences, BioPharmaceuticals R&D Unit, AstraZeneca, Gothenburg, Sweden.
  • Engberg S; Discovery Sciences, BioPharmaceuticals R&D Unit, AstraZeneca, Gothenburg, Sweden.
  • Maresca M; Discovery Sciences, BioPharmaceuticals R&D Unit, AstraZeneca, Gothenburg, Sweden.
  • Rooyackers O; Department of Clinical Sciences Intervention and Technology, Karolinska Institutet, Stockholm, Sweden.
  • Thöny B; Division of Metabolism and Children's Research Center, University Children's Hospital, Zürich, Switzerland.
  • Häberle J; Division of Metabolism and Children's Research Center, University Children's Hospital, Zürich, Switzerland.
  • Rosen B; Discovery Sciences, BioPharmaceuticals R&D Unit, AstraZeneca, Cambridge, UK.
  • Strom SC; Department of Laboratory Medicine, Karolinska Institutet, 141 52 Huddinge, Sweden. Electronic address: stephen.strom7.4@gmail.com.
Mol Ther ; 29(5): 1903-1917, 2021 05 05.
Article em En | MEDLINE | ID: mdl-33484963
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Ornitina Carbamoiltransferase / Doença da Deficiência de Ornitina Carbomoiltransferase / Hepatócitos / Edição de Genes / Mutação Limite: Adult / Aged / Animals / Child / Female / Humans / Male Idioma: En Revista: Mol Ther Ano de publicação: 2021 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Ornitina Carbamoiltransferase / Doença da Deficiência de Ornitina Carbomoiltransferase / Hepatócitos / Edição de Genes / Mutação Limite: Adult / Aged / Animals / Child / Female / Humans / Male Idioma: En Revista: Mol Ther Ano de publicação: 2021 Tipo de documento: Article