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Restoration of functional PAX6 in aniridia patient iPSC-derived ocular tissue models using repurposed nonsense suppression drugs.
Lima Cunha, Dulce; Sarkar, Hajrah; Eintracht, Jonathan; Harding, Philippa; Zhou, Jo Huiqing; Moosajee, Mariya.
Afiliação
  • Lima Cunha D; UCL Institute of Ophthalmology, London, UK.
  • Sarkar H; Radboud Institute of Molecular Life Sciences, Radboud University, Nijmegen, the Netherlands.
  • Eintracht J; UCL Institute of Ophthalmology, London, UK.
  • Harding P; The Francis Crick Institute, London, UK.
  • Zhou JH; UCL Institute of Ophthalmology, London, UK.
  • Moosajee M; UCL Institute of Ophthalmology, London, UK.
Mol Ther Nucleic Acids ; 33: 240-253, 2023 Sep 12.
Article em En | MEDLINE | ID: mdl-37483273

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Nucleic Acids Ano de publicação: 2023 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Nucleic Acids Ano de publicação: 2023 Tipo de documento: Article