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Novel bicistronic lentiviral vectors correct ß-Hexosaminidase deficiency in neural and hematopoietic stem cells and progeny: implications for in vivo and ex vivo gene therapy of GM2 gangliosidosis.
Ornaghi, Francesca; Sala, Davide; Tedeschi, Fabiana; Maffia, Maria Chiara; Bazzucchi, Martina; Morena, Francesco; Valsecchi, Manuela; Aureli, Massimo; Martino, Sabata; Gritti, Angela.
Afiliación
  • Ornaghi F; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Via Olgettina 60, 20132 Milan, Italy.
  • Sala D; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Via Olgettina 60, 20132 Milan, Italy; Department of Chemistry, Biology and Biotechnology, University of Perugia, Via del Giochetto, 06123 Perugia, Italy.
  • Tedeschi F; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Via Olgettina 60, 20132 Milan, Italy.
  • Maffia MC; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Via Olgettina 60, 20132 Milan, Italy.
  • Bazzucchi M; Department of Chemistry, Biology and Biotechnology, University of Perugia, Via del Giochetto, 06123 Perugia, Italy.
  • Morena F; Department of Chemistry, Biology and Biotechnology, University of Perugia, Via del Giochetto, 06123 Perugia, Italy.
  • Valsecchi M; Department of Medical Biotechnology and Translational Medicine, University of Milano, Via Fratelli Cervi 93, 20090 Segrate, MI, Italy.
  • Aureli M; Department of Medical Biotechnology and Translational Medicine, University of Milano, Via Fratelli Cervi 93, 20090 Segrate, MI, Italy.
  • Martino S; Department of Chemistry, Biology and Biotechnology, University of Perugia, Via del Giochetto, 06123 Perugia, Italy.
  • Gritti A; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Via Olgettina 60, 20132 Milan, Italy. Electronic address: gritti.angela@hsr.it.
Neurobiol Dis ; 134: 104667, 2020 02.
Article en En | MEDLINE | ID: mdl-31682993
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Células Madre Hematopoyéticas / Terapia Genética / Gangliosidosis GM2 / Cadena alfa de beta-Hexosaminidasa / Cadena beta de beta-Hexosaminidasa / Células-Madre Neurales Idioma: En Revista: Neurobiol Dis Asunto de la revista: NEUROLOGIA Año: 2020 Tipo del documento: Article
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Células Madre Hematopoyéticas / Terapia Genética / Gangliosidosis GM2 / Cadena alfa de beta-Hexosaminidasa / Cadena beta de beta-Hexosaminidasa / Células-Madre Neurales Idioma: En Revista: Neurobiol Dis Asunto de la revista: NEUROLOGIA Año: 2020 Tipo del documento: Article