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Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome.
Sereni, Lucia; Castiello, Maria Carmina; Di Silvestre, Dario; Della Valle, Patrizia; Brombin, Chiara; Ferrua, Francesca; Cicalese, Maria Pia; Pozzi, Loris; Migliavacca, Maddalena; Bernardo, Maria Ester; Pignata, Claudio; Farah, Roula; Notarangelo, Lucia Dora; Marcus, Nufar; Cattaneo, Lorella; Spinelli, Marco; Giannelli, Stefania; Bosticardo, Marita; van Rossem, Koen; D'Angelo, Armando; Aiuti, Alessandro; Mauri, Pierluigi; Villa, Anna.
  • Sereni 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.
  • Castiello MC; 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 Silvestre D; Proteomic and Metabolomic Laboratory, Institute of Biomedical Technologies, National Research Council (ITB-CNR), Segrate, Italy.
  • Della Valle P; Coagulation Service & Thrombosis Research Unit, San Raffaele Scientific Institute, Milan, Italy.
  • Brombin C; University Centre for Statistics in the Biomedical Sciences (CUSSB), Vita-Salute San Raffaele University, Milan, Italy.
  • Ferrua 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; Vita-Salute San Raffaele University, Milan, Italy; Pediatric Immunohematology Unit, San Raffaele Scientific Institute, M
  • Cicalese MP; 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, San Raffaele Scientific Institute, Milan, Italy.
  • Pozzi L; Coagulation Service & Thrombosis Research Unit, San Raffaele Scientific Institute, Milan, Italy.
  • Migliavacca 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; Pediatric Immunohematology Unit, San Raffaele Scientific Institute, Milan, Italy.
  • Bernardo ME; 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, San Raffaele Scientific Institute, Milan, Italy.
  • Pignata C; Pediatric Section, Department of Translational Medical Sciences, University of Naples Federico II, Naples, Italy.
  • Farah R; Department of Pediatrics, Division of Hematology-Oncology, Saint George Hospital University Medical Centre, Beirut, Lebanon.
  • Notarangelo LD; Pediatric Onco-Haematology and BMT Unit, Children's Hospital, ASST Spedali Civili of Brescia, Brescia, Italy.
  • Marcus N; Department of Pediatrics, Schneider Children's Medical Center of Israel, Petach Tikva, Israel; Kipper Institute of Immunology, Schneider Children's Medical Center of Israel, Petach Tikva, Israel; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
  • Cattaneo L; SC Pediatria, Ospedale Infantile C. Arrigo, Alessandria, Italy.
  • Spinelli M; Pediatric Clinic, MBBM Foundation, Maria Letizia Verga Center, Monza, Italy.
  • Giannelli 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.
  • Bosticardo 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.
  • van Rossem K; Rare Diseases Unit, GlaxoSmithKline, Brentford, United Kingdom.
  • D'Angelo A; Coagulation Service & Thrombosis Research Unit, San Raffaele Scientific Institute, 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; Vita-Salute San Raffaele University, Milan, Italy; Pediatric Immunohematology Unit, San Raffaele Scientific Institute, M
  • Mauri P; Proteomic and Metabolomic Laboratory, Institute of Biomedical Technologies, National Research Council (ITB-CNR), Segrate, Italy.
  • Villa 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; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy. Electronic addre
J Allergy Clin Immunol ; 144(3): 825-838, 2019 09.
Article en En | MEDLINE | ID: mdl-30926529
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Síndrome de Wiskott-Aldrich / Plaquetas / Terapia Genética / Lentivirus Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Año: 2019 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Search on Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Síndrome de Wiskott-Aldrich / Plaquetas / Terapia Genética / Lentivirus Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Año: 2019 Tipo del documento: Article