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Molecular and functional correction of a deep intronic splicing mutation in CFTR by CRISPR-Cas9 gene editing.
Walker, Amy J; Graham, Carina; Greenwood, Miriam; Woodall, Maximillian; Maeshima, Ruhina; O'Hara-Wright, Michelle; Sanz, David J; Guerrini, Ileana; Aldossary, Ahmad M; O'Callaghan, Christopher; Baines, Deborah L; Harrison, Patrick T; Hart, Stephen L.
Afiliação
  • Walker AJ; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.
  • Graham C; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.
  • Greenwood M; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.
  • Woodall M; Institute for Infection and Immunity, St. George's, University of London, London, UK.
  • Maeshima R; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.
  • O'Hara-Wright M; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.
  • Sanz DJ; Department of Physiology, BioSciences Institute, University College Cork, Cork, Ireland.
  • Guerrini I; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.
  • Aldossary AM; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.
  • O'Callaghan C; Infection, Immunity & Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK.
  • Baines DL; Institute for Infection and Immunity, St. George's, University of London, London, UK.
  • Harrison PT; Department of Physiology, BioSciences Institute, University College Cork, Cork, Ireland.
  • Hart SL; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.
Mol Ther Methods Clin Dev ; 31: 101140, 2023 Dec 14.
Article em En | MEDLINE | ID: mdl-38027060
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Texto completo: 1 Base de dados: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Reino Unido
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Reino Unido