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Paired nicking-mediated COL17A1 reframing for junctional epidermolysis bullosa.
Bischof, Johannes; March, Oliver Patrick; Liemberger, Bernadette; Haas, Simone Alexandra; Hainzl, Stefan; Petkovic, Igor; Leb-Reichl, Victoria; Illmer, Julia; Korotchenko, Evgeniia; Klausegger, Alfred; Hoog, Anna; Binder, Heide-Marie; Garcia, Marta; Duarte, Blanca; Strunk, Dirk; Larcher, Fernando; Reichelt, Julia; Guttmann-Gruber, Christina; Wally, Verena; Hofbauer, Josefina Piñón; Bauer, Johann Wolfgang; Cathomen, Toni; Kocher, Thomas; Koller, Ulrich.
Afiliação
  • Bischof J; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria.
  • March OP; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria.
  • Liemberger B; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria.
  • Haas SA; Institute for Transfusion Medicine and Gene Therapy, Medical Center - University of Freiburg, Freiburg, Germany; Center for Chronic Immunodeficiency, Medical Center - University of Freiburg, Freiburg, Germany.
  • Hainzl S; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria.
  • Petkovic I; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria.
  • Leb-Reichl V; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria.
  • Illmer J; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria.
  • Korotchenko E; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria.
  • Klausegger A; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria.
  • Hoog A; Cell Therapy Institute, SCI-TReCS, Paracelsus Medical University, Salzburg, Austria.
  • Binder HM; Cell Therapy Institute, SCI-TReCS, Paracelsus Medical University, Salzburg, Austria.
  • Garcia M; Epithelial Biomedicine Division, CIEMAT-CIBERER, Department of Bioengineering, UC3M, Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz, 28040 Madrid, Spain.
  • Duarte B; Epithelial Biomedicine Division, CIEMAT-CIBERER, Department of Bioengineering, UC3M, Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz, 28040 Madrid, Spain.
  • Strunk D; Cell Therapy Institute, SCI-TReCS, Paracelsus Medical University, Salzburg, Austria.
  • Larcher F; Epithelial Biomedicine Division, CIEMAT-CIBERER, Department of Bioengineering, UC3M, Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz, 28040 Madrid, Spain.
  • Reichelt J; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria.
  • Guttmann-Gruber C; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria.
  • Wally V; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria.
  • Hofbauer JP; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria.
  • Bauer JW; Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, 5020 Salzburg, Austria.
  • Cathomen T; Institute for Transfusion Medicine and Gene Therapy, Medical Center - University of Freiburg, Freiburg, Germany; Center for Chronic Immunodeficiency, Medical Center - University of Freiburg, Freiburg, Germany; Faculty of Medicine, University of Freiburg, Freiburg, Germany.
  • Kocher T; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria.
  • Koller U; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Müllner Hauptstraße 48, 5020 Salzburg, Austria. Electronic address: u.koller@salk.at.
Mol Ther ; 30(8): 2680-2692, 2022 08 03.
Article em En | MEDLINE | ID: mdl-35490295
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Autoantígenos / Epidermólise Bolhosa Juncional / Epidermólise Bolhosa / Colágenos não Fibrilares Limite: Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Áustria País de publicação: Estados Unidos
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Autoantígenos / Epidermólise Bolhosa Juncional / Epidermólise Bolhosa / Colágenos não Fibrilares Limite: Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Áustria País de publicação: Estados Unidos