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Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.
Fumagalli, Francesca; Calbi, Valeria; Natali Sora, Maria Grazia; Sessa, Maria; Baldoli, Cristina; Rancoita, Paola Maria V; Ciotti, Francesca; Sarzana, Marina; Fraschini, Maddalena; Zambon, Alberto Andrea; Acquati, Serena; Redaelli, Daniela; Attanasio, Vanessa; Miglietta, Simona; De Mattia, Fabiola; Barzaghi, Federica; Ferrua, Francesca; Migliavacca, Maddalena; Tucci, Francesca; Gallo, Vera; Del Carro, Ubaldo; Canale, Sabrina; Spiga, Ivana; Lorioli, Laura; Recupero, Salvatore; Fratini, Elena Sophia; Morena, Francesco; Silvani, Paolo; Calvi, Maria Rosa; Facchini, Marcella; Locatelli, Sara; Corti, Ambra; Zancan, Stefano; Antonioli, Gigliola; Farinelli, Giada; Gabaldo, Michela; Garcia-Segovia, Jesus; Schwab, Laetitia C; Downey, Gerald F; Filippi, Massimo; Cicalese, Maria Pia; Martino, Sabata; Di Serio, Clelia; Ciceri, Fabio; Bernardo, Maria Ester; Naldini, Luigi; Biffi, Alessandra; Aiuti, Alessandro.
Afiliação
  • Fumagalli F; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy; Units of Neurology and Neurophysiology, IRCCS San Raffaele Scientific Institute,
  • Calbi V; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Natali Sora MG; Units of Neurology and Neurophysiology, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Sessa M; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Department of Neurology, ASST Papa Giovanni XXIII Bergamo, Italy.
  • Baldoli C; Neuroradiology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Rancoita PMV; University Centre of Statistics in the Biomedical Sciences (CUSSB), Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy.
  • Ciotti F; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Sarzana M; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Fraschini M; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Zambon AA; Units of Neurology and Neurophysiology, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Acquati S; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Redaelli D; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Attanasio V; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Miglietta S; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • De Mattia F; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Barzaghi F; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Ferrua F; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Migliavacca M; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Tucci F; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Gallo V; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Del Carro U; Units of Neurology and Neurophysiology, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Canale S; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Department of Specialistic Neurological Rehabilitation, IRCCS Multimedica, Sesto San Giovanni, Italy.
  • Spiga I; Clinical Molecular Biology Laboratory, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Lorioli L; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Recupero S; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy.
  • Fratini ES; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy.
  • Morena F; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Department of Chemistry, Biology and Biotechnologies, University of Perugia, Perugia, Italy.
  • Silvani P; Department of Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Calvi MR; Department of Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Facchini M; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Locatelli S; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Corti A; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Zancan S; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Antonioli G; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Farinelli G; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Gabaldo M; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Garcia-Segovia J; Orchard Therapeutics (Europe), London, UK.
  • Schwab LC; Orchard Therapeutics (Europe), London, UK.
  • Downey GF; Orchard Therapeutics (Europe), London, UK.
  • Filippi M; Units of Neurology and Neurophysiology, IRCCS San Raffaele Scientific Institute, Milan, Italy; Unit of Neurorehabilitation, IRCCS San Raffaele Scientific Institute, Milan, Italy; Neuroimaging Research Unit, Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute
  • Cicalese MP; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Martino S; Department of Chemistry, Biology and Biotechnologies, University of Perugia, Perugia, Italy.
  • Di Serio C; University Centre of Statistics in the Biomedical Sciences (CUSSB), Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy; Biomedical Faculty, Università della Svizzera Italiana, Lugano, Switzerland.
  • Ciceri F; Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy.
  • Bernardo ME; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy.
  • Naldini L; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), 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), IRCCS San Raffaele Scientific Institute, Milan, Italy; Division of Pediatric Hematology, Oncology and Stem Cell Transplant, Padua University and Padua University Hospital, Padua, Italy; Gene Therapy Program, Dana Farber/Boston Children's C
  • Aiuti A; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy. Electronic address: aiuti.ale
Lancet ; 399(10322): 372-383, 2022 01 22.
Article em En | MEDLINE | ID: mdl-35065785
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Cerebrosídeo Sulfatase / Lentivirus / Transplante de Células-Tronco Hematopoéticas / Leucodistrofia Metacromática Tipo de estudo: Clinical_trials / Observational_studies / Prognostic_studies Limite: Child / Child, preschool / Female / Humans / Male País/Região como assunto: Europa Idioma: En Revista: Lancet Ano de publicação: 2022 Tipo de documento: Article
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Cerebrosídeo Sulfatase / Lentivirus / Transplante de Células-Tronco Hematopoéticas / Leucodistrofia Metacromática Tipo de estudo: Clinical_trials / Observational_studies / Prognostic_studies Limite: Child / Child, preschool / Female / Humans / Male País/Região como assunto: Europa Idioma: En Revista: Lancet Ano de publicação: 2022 Tipo de documento: Article