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Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial.
Sessa, Maria; Lorioli, Laura; Fumagalli, Francesca; Acquati, Serena; Redaelli, Daniela; Baldoli, Cristina; Canale, Sabrina; Lopez, Ignazio D; Morena, Francesco; Calabria, Andrea; Fiori, Rossana; Silvani, Paolo; Rancoita, Paola M V; Gabaldo, Michela; Benedicenti, Fabrizio; Antonioli, Gigliola; Assanelli, Andrea; Cicalese, Maria Pia; Del Carro, Ubaldo; Sora, Maria Grazia Natali; Martino, Sabata; Quattrini, Angelo; Montini, Eugenio; Di Serio, Clelia; Ciceri, Fabio; Roncarolo, Maria Grazia; Aiuti, Alessandro; Naldini, Luigi; Biffi, Alessandra.
Afiliación
  • Sessa M; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy; Neurology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Lorioli L; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; Bone Marrow Transplantation Prog
  • Fumagalli F; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy; Neurology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Acquati S; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Redaelli D; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Baldoli C; Neuroradiology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Canale S; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Lopez ID; Neurology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Morena F; Department of Chemistry, Biology and Biotechnologies, University of Perugia, Perugia, Italy.
  • Calabria A; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Fiori R; Department of Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Silvani P; Department of Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Rancoita PM; Vita Salute San Raffaele University, Milan, Italy.
  • Gabaldo M; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Benedicenti F; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Antonioli G; Pediatric Immunohematology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; Bone Marrow Transplantation Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Assanelli A; Pediatric Immunohematology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; Bone Marrow Transplantation Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Cicalese MP; Pediatric Immunohematology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; Bone Marrow Transplantation Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Del Carro U; Neurology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Sora MG; Neurology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Martino S; Department of Chemistry, Biology and Biotechnologies, University of Perugia, Perugia, Italy.
  • Quattrini A; Neurology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Montini E; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Di Serio C; Vita Salute San Raffaele University, Milan, Italy.
  • Ciceri F; Bone Marrow Transplantation Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Roncarolo MG; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita Salute San Raffaele University, Milan, Italy.
  • Aiuti A; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; Bone Marrow Transplantation Prog
  • Naldini L; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita Salute San Raffaele University, Milan, Italy.
  • Biffi A; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; Bone Marrow Transplantation Prog
Lancet ; 388(10043): 476-87, 2016 Jul 30.
Article en En | MEDLINE | ID: mdl-27289174
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Trasplante de Células Madre Hematopoyéticas / Leucodistrofia Metacromática Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Adolescent / Child / Child, preschool / Female / Humans / Infant / Male País/Región como asunto: Europa Idioma: En Revista: Lancet Año: 2016 Tipo del documento: Article País de afiliación: Italia
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Trasplante de Células Madre Hematopoyéticas / Leucodistrofia Metacromática Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Adolescent / Child / Child, preschool / Female / Humans / Infant / Male País/Región como asunto: Europa Idioma: En Revista: Lancet Año: 2016 Tipo del documento: Article País de afiliación: Italia