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Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy.
Ricca, Alessandra; Rufo, Nicole; Ungari, Silvia; Morena, Francesco; Martino, Sabata; Kulik, Wilem; Alberizzi, Valeria; Bolino, Alessandra; Bianchi, Francesca; Del Carro, Ubaldo; Biffi, Alessandra; Gritti, Angela.
Afiliación
  • Ricca A; San Raffaele Scientific Institute, Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Telethon Institute for Gene Therapy (TIGET), Via Olgettina 58, Milano 20132, Italy.
  • Rufo N; San Raffaele Scientific Institute, Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Telethon Institute for Gene Therapy (TIGET), Via Olgettina 58, Milano 20132, Italy.
  • Ungari S; San Raffaele Scientific Institute, Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Telethon Institute for Gene Therapy (TIGET), Via Olgettina 58, Milano 20132, Italy.
  • Morena F; Department of Chemistry, Biology and Biotechnologies, University of Perugia, via del Giochetto, Perugia, Italy.
  • Martino S; Department of Chemistry, Biology and Biotechnologies, University of Perugia, via del Giochetto, Perugia, Italy.
  • Kulik W; Laboratory of Genetic Metabolic Diseases, Academic Medical Center AMC, University of Amsterdam, Meibergdreef 9, Amsterdam, The Netherlands and.
  • Alberizzi V; Division of Neuroscience, San Raffaele Scientific Institute, INSPE, Via Olgettina 58, Milano, Italy.
  • Bolino A; Division of Neuroscience, San Raffaele Scientific Institute, INSPE, Via Olgettina 58, Milano, Italy.
  • Bianchi F; Division of Neuroscience, San Raffaele Scientific Institute, INSPE, Via Olgettina 58, Milano, Italy.
  • Del Carro U; Division of Neuroscience, San Raffaele Scientific Institute, INSPE, Via Olgettina 58, Milano, Italy.
  • Biffi A; San Raffaele Scientific Institute, Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Telethon Institute for Gene Therapy (TIGET), Via Olgettina 58, Milano 20132, Italy.
  • Gritti A; San Raffaele Scientific Institute, Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Telethon Institute for Gene Therapy (TIGET), Via Olgettina 58, Milano 20132, Italy, gritti.angela@hsr.it.
Hum Mol Genet ; 24(12): 3372-89, 2015 Jun 15.
Article en En | MEDLINE | ID: mdl-25749991
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Terapia Genética / Tratamiento Basado en Trasplante de Células y Tejidos / Galactosilceramidasa / Leucodistrofia de Células Globoides Tipo de estudio: Diagnostic_studies / Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Hum Mol Genet Asunto de la revista: BIOLOGIA MOLECULAR / GENETICA MEDICA Año: 2015 Tipo del documento: Article País de afiliación: Italia
Texto completo
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Terapia Genética / Tratamiento Basado en Trasplante de Células y Tejidos / Galactosilceramidasa / Leucodistrofia de Células Globoides Tipo de estudio: Diagnostic_studies / Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Hum Mol Genet Asunto de la revista: BIOLOGIA MOLECULAR / GENETICA MEDICA Año: 2015 Tipo del documento: Article País de afiliación: Italia