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Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice.
Padula, Agnese; Petruzzelli, Raffaella; Philbert, Sasha A; Church, Stephanie J; Esposito, Federica; Campione, Severo; Monti, Marcello; Capolongo, Filomena; Perna, Claudia; Nusco, Edoardo; Schmidt, Hartmut H; Auricchio, Alberto; Cooper, Garth J S; Polishchuk, Roman; Piccolo, Pasquale.
Afiliación
  • Padula A; Telethon Institute of Genetics and Medicine, Pozzuoli, Italy.
  • Petruzzelli R; Telethon Institute of Genetics and Medicine, Pozzuoli, Italy.
  • Philbert SA; Scuola Superiore Meridionale, University of Naples Federico II, Naples, Italy.
  • Church SJ; Division of Cardiovascular Sciences, School of Medical Sciences, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, UK.
  • Esposito F; Centre for Advanced Discovery and Experimental Therapeutics (CADET), Manchester Academic Health Sciences Centre, Manchester, UK.
  • Campione S; Division of Cardiovascular Sciences, School of Medical Sciences, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, UK.
  • Monti M; Centre for Advanced Discovery and Experimental Therapeutics (CADET), Manchester Academic Health Sciences Centre, Manchester, UK.
  • Capolongo F; Telethon Institute of Genetics and Medicine, Pozzuoli, Italy.
  • Perna C; Pathology Unit, A. Cardarelli Hospital, Naples, Italy.
  • Nusco E; Telethon Institute of Genetics and Medicine, Pozzuoli, Italy.
  • Schmidt HH; Telethon Institute of Genetics and Medicine, Pozzuoli, Italy.
  • Auricchio A; Telethon Institute of Genetics and Medicine, Pozzuoli, Italy.
  • Cooper GJS; Telethon Institute of Genetics and Medicine, Pozzuoli, Italy.
  • Polishchuk R; Department of Gastroenterology and Hepatology, University Hospital Duisburg-Essen, Essen, Germany.
  • Piccolo P; Telethon Institute of Genetics and Medicine, Pozzuoli, Italy.
Mol Ther Methods Clin Dev ; 26: 495-504, 2022 Sep 08.
Article en En | MEDLINE | ID: mdl-36092366

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Año: 2022 Tipo del documento: Article País de afiliación: Italia

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Año: 2022 Tipo del documento: Article País de afiliación: Italia