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QR-313, an Antisense Oligonucleotide, Shows Therapeutic Efficacy for Treatment of Dominant and Recessive Dystrophic Epidermolysis Bullosa: A Preclinical Study.
Bornert, Olivier; Hogervorst, Marieke; Nauroy, Pauline; Bischof, Johannes; Swildens, Jim; Athanasiou, Ioannis; Tufa, Sara F; Keene, Douglas R; Kiritsi, Dimitra; Hainzl, Stefan; Murauer, Eva M; Marinkovich, M Peter; Platenburg, Gerard; Hausser, Ingrid; Wally, Verena; Ritsema, Tita; Koller, Ulrich; Haisma, Elisabeth M; Nyström, Alexander.
Afiliação
  • Bornert O; Department of Dermatology, Medical Faculty, Medical Center - University of Freiburg, Freiburg, Germany.
  • Hogervorst M; ProQR Therapeutics N.V., Leiden, The Netherlands.
  • Nauroy P; Department of Dermatology, Medical Faculty, Medical Center - University of Freiburg, Freiburg, Germany.
  • 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, Salzburg, Austria.
  • Swildens J; ProQR Therapeutics N.V., Leiden, The Netherlands.
  • Athanasiou I; Department of Dermatology, Medical Faculty, Medical Center - University of Freiburg, Freiburg, Germany.
  • Tufa SF; Micro-Imaging Center, Shriners Hospital for Children, Portland, Oregon, USA.
  • Keene DR; Micro-Imaging Center, Shriners Hospital for Children, Portland, Oregon, USA.
  • Kiritsi D; Department of Dermatology, Medical Faculty, 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, Salzburg, Austria.
  • Murauer EM; EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University Salzburg, Salzburg, Austria.
  • Marinkovich MP; Department of Dermatology, Stanford University School of Medicine, Stanford, California, USA; Dermatology, Veteran's Affairs Medical Center, Palo Alto, California, USA.
  • Platenburg G; ProQR Therapeutics N.V., Leiden, The Netherlands.
  • Hausser I; Institute of Pathology, Heidelberg University Hospital, Heidelberg, Germany.
  • 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, Salzburg, Austria.
  • Ritsema T; ProQR Therapeutics N.V., Leiden, The Netherlands.
  • 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, Salzburg, Austria.
  • Haisma EM; ProQR Therapeutics N.V., Leiden, The Netherlands.
  • Nyström A; Department of Dermatology, Medical Faculty, Medical Center - University of Freiburg, Freiburg, Germany. Electronic address: alexander.nystroem@uniklinik-freiburg.de.
J Invest Dermatol ; 141(4): 883-893.e6, 2021 04.
Article em En | MEDLINE | ID: mdl-32946877
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Cicatrização / Terapia Genética / Epidermólise Bolhosa Distrófica / Oligonucleotídeos Antissenso / Colágeno Tipo VII Limite: Animals / Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Cicatrização / Terapia Genética / Epidermólise Bolhosa Distrófica / Oligonucleotídeos Antissenso / Colágeno Tipo VII Limite: Animals / Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article