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Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study.
Ferrua, Francesca; Cicalese, Maria Pia; Galimberti, Stefania; Giannelli, Stefania; Dionisio, Francesca; Barzaghi, Federica; Migliavacca, Maddalena; Bernardo, Maria Ester; Calbi, Valeria; Assanelli, Andrea Angelo; Facchini, Marcella; Fossati, Claudia; Albertazzi, Elena; Scaramuzza, Samantha; Brigida, Immacolata; Scala, Serena; Basso-Ricci, Luca; Pajno, Roberta; Casiraghi, Miriam; Canarutto, Daniele; Salerio, Federica Andrea; Albert, Michael H; Bartoli, Antonella; Wolf, Hermann M; Fiori, Rossana; Silvani, Paolo; Gattillo, Salvatore; Villa, Anna; Biasco, Luca; Dott, Christopher; Culme-Seymour, Emily J; van Rossem, Koenraad; Atkinson, Gillian; Valsecchi, Maria Grazia; Roncarolo, Maria Grazia; Ciceri, Fabio; Naldini, Luigi; Aiuti, Alessandro.
Affiliation
  • Ferrua F; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy.
  • Cicalese MP; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Galimberti S; Center of Biostatistics for Clinical Epidemiology, University of Milano-Bicocca, Monza, Italy.
  • Giannelli S; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Dionisio 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 and Bone Marrow Transplantation Unit, 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 and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Bernardo ME; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Calbi V; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Assanelli AA; Hematology and Bone Marrow Transplantation Unit, 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.
  • Fossati C; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Albertazzi E; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Scaramuzza S; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Brigida I; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Scala S; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Basso-Ricci L; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Pajno R; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy.
  • Casiraghi M; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Canarutto D; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy.
  • Salerio FA; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Albert MH; Department of Pediatric Hematology/Oncology, Dr von Haunersches University Children's Hospital, Munich, Germany.
  • Bartoli A; Clinical Pharmacokinetics Unit, San Matteo Hospital, Pavia, Italy.
  • Wolf HM; Immunology Outpatient Clinic, and Sigmund Freud Private University-Medical School, Vienna, Austria.
  • 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.
  • Gattillo S; Blood Transfusion Service, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  • Villa A; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.
  • Biasco L; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; University College London, Great Ormond Street Institute of Child Health, Faculty of Population Health Sciences, London, UK.
  • Dott C; CSD Pharma Consulting, Redhill, UK; Orchard Therapeutics, London, UK.
  • Culme-Seymour EJ; Rare Diseases Unit, GlaxoSmithKline, Brentford, UK; Sangamo Therapeutics, London, UK.
  • van Rossem K; Rare Diseases Unit, GlaxoSmithKline, Brentford, UK.
  • Atkinson G; Rare Diseases Unit, GlaxoSmithKline, Brentford, UK; Sangamo Therapeutics, London, UK.
  • Valsecchi MG; Center of Biostatistics for Clinical Epidemiology, University of Milano-Bicocca, Monza, Italy.
  • Roncarolo MG; Division of Stem Cell Transplantation and Regenerative Medicine and Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, CA, USA.
  • Ciceri F; Hematology and Bone Marrow Transplantation Unit, 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.
  • Aiuti A; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy. Electronic ad
Lancet Haematol ; 6(5): e239-e253, 2019 May.
Article in En | MEDLINE | ID: mdl-30981783
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Wiskott-Aldrich Syndrome / Hematopoietic Stem Cells / Genetic Therapy / Lentivirus / Genetic Vectors Type of study: Clinical_trials / Diagnostic_studies Limits: Child / Child, preschool / Female / Humans / Infant / Male Country/Region as subject: Europa Language: En Journal: Lancet Haematol Year: 2019 Document type: Article
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Wiskott-Aldrich Syndrome / Hematopoietic Stem Cells / Genetic Therapy / Lentivirus / Genetic Vectors Type of study: Clinical_trials / Diagnostic_studies Limits: Child / Child, preschool / Female / Humans / Infant / Male Country/Region as subject: Europa Language: En Journal: Lancet Haematol Year: 2019 Document type: Article