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Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa.
Fischer, M Dominik; McClements, Michelle E; Martinez-Fernandez de la Camara, Cristina; Bellingrath, Julia-Sophia; Dauletbekov, Daniyar; Ramsden, Simon C; Hickey, Doron G; Barnard, Alun R; MacLaren, Robert E.
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  • Fischer MD; Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, The John Radcliffe Hospital, Levels 5 & 6, West Wing, Headley Way, OX3 9DU Oxford, UK; University Eye Hospital, Center for Opthalmology, Elfriede-Aulhorn-Strasse 7, 72076 Tübingen, Germany.
  • McClements ME; Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, The John Radcliffe Hospital, Levels 5 & 6, West Wing, Headley Way, OX3 9DU Oxford, UK.
  • Martinez-Fernandez de la Camara C; Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, The John Radcliffe Hospital, Levels 5 & 6, West Wing, Headley Way, OX3 9DU Oxford, UK.
  • Bellingrath JS; Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, The John Radcliffe Hospital, Levels 5 & 6, West Wing, Headley Way, OX3 9DU Oxford, UK; University Eye Hospital, Center for Opthalmology, Elfriede-Aulhorn-Strasse 7, 72076 Tübingen, Germany.
  • Dauletbekov D; Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, The John Radcliffe Hospital, Levels 5 & 6, West Wing, Headley Way, OX3 9DU Oxford, UK; University Eye Hospital, Center for Opthalmology, Elfriede-Aulhorn-Strasse 7, 72076 Tübingen, Germany.
  • Ramsden SC; Centre for Genomic Medicine, Central Manchester University Hospitals NHS Foundation Trust, Manchester Academic Health Sciences Centre, M13 9WL Manchester, UK.
  • Hickey DG; Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, The John Radcliffe Hospital, Levels 5 & 6, West Wing, Headley Way, OX3 9DU Oxford, UK.
  • Barnard AR; Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, The John Radcliffe Hospital, Levels 5 & 6, West Wing, Headley Way, OX3 9DU Oxford, UK.
  • MacLaren RE; Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, The John Radcliffe Hospital, Levels 5 & 6, West Wing, Headley Way, OX3 9DU Oxford, UK; Oxford Eye Hospital, Oxford University Hospitals NHS Trust, The John Radcliffe Hospital, West Wing, Headley Way, OX3 9DU Oxford, UK.
Mol Ther ; 25(8): 1854-1865, 2017 08 02.
Article en En | MEDLINE | ID: mdl-28549772
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Codón / Terapia Genética / Proteínas Portadoras / Retinitis Pigmentosa / Dependovirus / Genes Ligados a X / Proteínas del Ojo / Vectores Genéticos Tipo de estudio: Diagnostic_studies / Prognostic_studies Límite: Animals Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2017 Tipo del documento: Article País de afiliación: Alemania Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Codón / Terapia Genética / Proteínas Portadoras / Retinitis Pigmentosa / Dependovirus / Genes Ligados a X / Proteínas del Ojo / Vectores Genéticos Tipo de estudio: Diagnostic_studies / Prognostic_studies Límite: Animals Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2017 Tipo del documento: Article País de afiliación: Alemania Pais de publicación: Estados Unidos