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CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids.
Afanasyeva, Tess A V; Athanasiou, Dimitra; Perdigao, Pedro R L; Whiting, Kae R; Duijkers, Lonneke; Astuti, Galuh D N; Bennett, Jean; Garanto, Alejandro; van der Spuy, Jacqueline; Roepman, Ronald; Cheetham, Michael E; Collin, Rob W J.
Affiliation
  • Afanasyeva TAV; Department of Human Genetics, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands.
  • Athanasiou D; Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, 6525 GD Nijmegen, the Netherlands.
  • Perdigao PRL; UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
  • Whiting KR; UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
  • Duijkers L; Department of Human Genetics, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands.
  • Astuti GDN; Department of Human Genetics, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands.
  • Bennett J; Department of Human Genetics, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands.
  • Garanto A; Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia PA 19104, USA.
  • van der Spuy J; Department of Human Genetics, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands.
  • Roepman R; Department of Paediatrics, Amalia Children's Hospital, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands.
  • Cheetham ME; UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
  • Collin RWJ; Department of Human Genetics, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands.
Mol Ther Methods Clin Dev ; 29: 522-531, 2023 Jun 08.
Article in En | MEDLINE | ID: mdl-37305852

Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Ther Methods Clin Dev Year: 2023 Document type: Article

Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Ther Methods Clin Dev Year: 2023 Document type: Article