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Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency.
Cicalese, Maria Pia; Ferrua, Francesca; Castagnaro, Laura; Pajno, Roberta; Barzaghi, Federica; Giannelli, Stefania; Dionisio, Francesca; Brigida, Immacolata; Bonopane, Marco; Casiraghi, Miriam; Tabucchi, Antonella; Carlucci, Filippo; Grunebaum, Eyal; Adeli, Mehdi; Bredius, Robbert G; Puck, Jennifer M; Stepensky, Polina; Tezcan, Ilhan; Rolfe, Katie; De Boever, Erika; Reinhardt, Rickey R; Appleby, Jonathan; Ciceri, Fabio; Roncarolo, Maria Grazia; Aiuti, Alessandro.
Afiliación
  • Cicalese MP; San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy;
  • Ferrua F; San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy;
  • Castagnaro L; San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy;
  • Pajno R; Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy;
  • Barzaghi F; San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy;
  • Giannelli S; San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy;
  • Dionisio F; San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy;
  • Brigida I; San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy;
  • Bonopane M; San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy;
  • Casiraghi M; San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy;
  • Tabucchi A; Department of Medical Biotechnology, University of Siena, Siena, Italy;
  • Carlucci F; Department of Medical Biotechnology, University of Siena, Siena, Italy;
  • Grunebaum E; Division of Immunology and Allergy, The Hospital for Sick Children, Toronto, Canada;
  • Adeli M; Division of Immunology and Allergy, Hamad Medical Corporation, Doha, Qatar;
  • Bredius RG; Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands;
  • Puck JM; Department of Pediatrics, University of California-San Francisco, San Francisco, CA;
  • Stepensky P; Pediatric Hematology-Oncology and Bone Marrow Transplantation, Hadassah Hebrew University Hospital, Jerusalem, Israel;
  • Tezcan I; Department of Pediatrics, Section of Pediatric Immunology, Hacettepe University Faculty of Medicine, Ankara, Turkey;
  • Rolfe K; GSK Research and Development, GlaxoSmithKline, Stevenage, United Kingdom, and Upper Merion, PA;
  • De Boever E; GSK Research and Development, GlaxoSmithKline, Stevenage, United Kingdom, and Upper Merion, PA;
  • Reinhardt RR; GSK Research and Development, GlaxoSmithKline, Stevenage, United Kingdom, and Upper Merion, PA;
  • Appleby J; GSK Research and Development, GlaxoSmithKline, Stevenage, United Kingdom, and Upper Merion, PA;
  • Ciceri F; Vita-Salute San Raffaele University, Milan, Italy; Hematology and Bone Marrow Transplant Unit, San Raffaele Scientific Institute, Milan, Italy; and.
  • Roncarolo MG; San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy; Department of Pediatrics Division of Stem Cell Transplantation and Regenerative Medicine, and Institute for Stem Cell Biology and Regenerative Medicin
  • Aiuti A; San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy; Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy;
Blood ; 128(1): 45-54, 2016 07 07.
Article en En | MEDLINE | ID: mdl-27129325
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Retroviridae / Terapia Genética / Adenosina Desaminasa / Inmunodeficiencia Combinada Grave / Recuperación de la Función / Agammaglobulinemia Tipo de estudio: Clinical_trials Límite: Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Blood Año: 2016 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
Añadir a Mi BVS
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XML
PubMed Links
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Retroviridae / Terapia Genética / Adenosina Desaminasa / Inmunodeficiencia Combinada Grave / Recuperación de la Función / Agammaglobulinemia Tipo de estudio: Clinical_trials Límite: Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Blood Año: 2016 Tipo del documento: Article Pais de publicación: Estados Unidos