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Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia: A Nonrandomized Controlled Trial.
Fischer, M Dominik; Michalakis, Stylianos; Wilhelm, Barbara; Zobor, Ditta; Muehlfriedel, Regine; Kohl, Susanne; Weisschuh, Nicole; Ochakovski, G Alex; Klein, Reinhild; Schoen, Christian; Sothilingam, Vithiyanjali; Garcia-Garrido, Marina; Kuehlewein, Laura; Kahle, Nadine; Werner, Annette; Dauletbekov, Daniyar; Paquet-Durand, François; Tsang, Stephen; Martus, Peter; Peters, Tobias; Seeliger, Mathias; Bartz-Schmidt, Karl Ulrich; Ueffing, Marius; Zrenner, Eberhart; Biel, Martin; Wissinger, Bernd.
Afiliação
  • Fischer MD; University Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Michalakis S; Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Wilhelm B; Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Zobor D; Center for Integrated Protein Science Munich, Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-University of Munich, Munich, Germany.
  • Muehlfriedel R; Department of Ophthalmology, Ludwig-Maximilians-Universität München, Munich, Germany.
  • Kohl S; Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Weisschuh N; Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Ochakovski GA; Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Klein R; Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Schoen C; Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Sothilingam V; University Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Garcia-Garrido M; Department of Internal Medicine II, University Hospital Tübingen, Germany.
  • Kuehlewein L; Center for Integrated Protein Science Munich, Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-University of Munich, Munich, Germany.
  • Kahle N; Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Werner A; Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Dauletbekov D; University Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Paquet-Durand F; University Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Tsang S; Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Martus P; Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Peters T; University Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Seeliger M; Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Bartz-Schmidt KU; Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Ueffing M; Department of Ophthalmology, College of Physicians and Surgeons, Columbia University, New York, New York.
  • Zrenner E; Institute for Clinical Epidemiology and Applied Biostatistics, University Hospital Tübingen, Tübingen, Germany.
  • Biel M; Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
  • Wissinger B; Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
JAMA Ophthalmol ; 138(6): 643-651, 2020 06 01.
Article em En | MEDLINE | ID: mdl-32352493
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Acuidade Visual / Defeitos da Visão Cromática / Células Fotorreceptoras Retinianas Cones / Canais de Cátion Regulados por Nucleotídeos Cíclicos Tipo de estudo: Diagnostic_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Adult / Female / Humans / Male / Middle aged Idioma: En Ano de publicação: 2020 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Acuidade Visual / Defeitos da Visão Cromática / Células Fotorreceptoras Retinianas Cones / Canais de Cátion Regulados por Nucleotídeos Cíclicos Tipo de estudo: Diagnostic_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Adult / Female / Humans / Male / Middle aged Idioma: En Ano de publicação: 2020 Tipo de documento: Article