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Combined RNA interference and gene replacement therapy targeting MFN2 as proof of principle for the treatment of Charcot-Marie-Tooth type 2A.
Rizzo, Federica; Bono, Silvia; Ruepp, Marc David; Salani, Sabrina; Ottoboni, Linda; Abati, Elena; Melzi, Valentina; Cordiglieri, Chiara; Pagliarani, Serena; De Gioia, Roberta; Anastasia, Alessia; Taiana, Michela; Garbellini, Manuela; Lodato, Simona; Kunderfranco, Paolo; Cazzato, Daniele; Cartelli, Daniele; Lonati, Caterina; Bresolin, Nereo; Comi, Giacomo; Nizzardo, Monica; Corti, Stefania.
Afiliación
  • Rizzo F; Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • Bono S; Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • Ruepp MD; United Kingdom Dementia Research Institute Centre, Institute of Psychiatry, Psychology and Neuroscience, King's College London, Maurice Wohl Clinical Neuroscience Institute, London, UK.
  • Salani S; Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • Ottoboni L; Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • Abati E; Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • Melzi V; Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • Cordiglieri C; Istituto Di Genetica Molecolare "Romeo Ed Enrica Invernizzi", Milan, Italy.
  • Pagliarani S; Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • De Gioia R; Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • Anastasia A; Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • Taiana M; Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • Garbellini M; Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • Lodato S; Department of Biomedical Sciences, Humanitas University, Pieve Emanuele, 20072, Milan, Italy.
  • Kunderfranco P; IRCCS Humanitas Research Hospital, Rozzano, 20089, Milan, Italy.
  • Cazzato D; Department of Biomedical Sciences, Humanitas University, Pieve Emanuele, 20072, Milan, Italy.
  • Cartelli D; IRCCS Humanitas Research Hospital, Rozzano, 20089, Milan, Italy.
  • Lonati C; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
  • Bresolin N; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
  • Comi G; Center for Preclinical Research, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via Pace 9, 20100, Milan, Italy.
  • Nizzardo M; Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • Corti S; Dino Ferrari Centre, Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), University of Milan, Milan, Italy.
Cell Mol Life Sci ; 80(12): 373, 2023 Nov 25.
Article en En | MEDLINE | ID: mdl-38007410
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Enfermedad de Charcot-Marie-Tooth / Células Madre Pluripotentes Inducidas Límite: Animals / Humans Idioma: En Revista: Cell Mol Life Sci Asunto de la revista: BIOLOGIA MOLECULAR Año: 2023 Tipo del documento: Article País de afiliación: Italia
Texto completo
Imprimir
XML
PubMed Links
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Enfermedad de Charcot-Marie-Tooth / Células Madre Pluripotentes Inducidas Límite: Animals / Humans Idioma: En Revista: Cell Mol Life Sci Asunto de la revista: BIOLOGIA MOLECULAR Año: 2023 Tipo del documento: Article País de afiliación: Italia