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AAV-mediated gene augmentation therapy of CRB1 patient-derived retinal organoids restores the histological and transcriptional retinal phenotype.
Boon, Nanda; Lu, Xuefei; Andriessen, Charlotte A; Moustakas, Ioannis; Buck, Thilo M; Freund, Christian; Arendzen, Christiaan H; Böhringer, Stefan; Mei, Hailiang; Wijnholds, Jan.
Afiliação
  • Boon N; Department of Ophthalmology, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
  • Lu X; Department of Ophthalmology, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
  • Andriessen CA; Department of Ophthalmology, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
  • Moustakas I; Sequencing Analysis Support Core, Department of Biomedical Data Sciences, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
  • Buck TM; Department of Ophthalmology, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
  • Freund C; hiPSC Hotel, Department of Anatomy and Embryology, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
  • Arendzen CH; hiPSC Hotel, Department of Anatomy and Embryology, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
  • Böhringer S; Department of Biomedical Data Sciences, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
  • Mei H; Sequencing Analysis Support Core, Department of Biomedical Data Sciences, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands.
  • Wijnholds J; Department of Ophthalmology, Leiden University Medical Center (LUMC), Albinusdreef 2, 2333 ZA Leiden, the Netherlands; Netherlands Institute for Neuroscience, an Institute of the Royal Netherlands Academy of Arts and Sciences (KNAW), Meibergdreef 47, 1105 BA Amsterdam, the Netherlands. Electronic ad
Stem Cell Reports ; 18(5): 1123-1137, 2023 05 09.
Article em En | MEDLINE | ID: mdl-37084726

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Proteínas de Membrana / Proteínas do Tecido Nervoso Idioma: En Ano de publicação: 2023 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Proteínas de Membrana / Proteínas do Tecido Nervoso Idioma: En Ano de publicação: 2023 Tipo de documento: Article