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Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety.
Cicalese, Maria Pia; Ferrua, Francesca; Castagnaro, Laura; Rolfe, Katie; De Boever, Erika; Reinhardt, Rickey R; Appleby, Jonathan; Roncarolo, Maria Grazia; Aiuti, Alessandro.
Afiliación
  • Cicalese MP; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy, 20132; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy, 20132.
  • Ferrua F; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy, 20132; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy, 20132; Vita-Salute San Raffaele University, Milan, Italy,
  • Castagnaro L; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy, 20132; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy, 20132.
  • Rolfe K; GSK Research and Development, GlaxoSmithKline, UB11 1BT and SG1 2NY, UK.
  • De Boever E; GSK Research and Development, GlaxoSmithKline, King of Prussia, PA 19406, USA.
  • Reinhardt RR; GSK Research and Development, GlaxoSmithKline, King of Prussia, PA 19406, USA.
  • Appleby J; GSK Research and Development, GlaxoSmithKline, UB11 1BT and SG1 2NY, UK.
  • Roncarolo MG; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy, 20132; Vita-Salute San Raffaele University, Milan, Italy, 20132; Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University, Stanfor
  • Aiuti A; San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy, 20132; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy, 20132; Vita-Salute San Raffaele University, Milan, Italy,
Mol Ther ; 26(3): 917-931, 2018 03 07.
Article en En | MEDLINE | ID: mdl-29433935
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Terapia Genética / Adenosina Desaminasa / Inmunodeficiencia Combinada Grave / Agammaglobulinemia Tipo de estudio: Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Child / Child, preschool / Humans / Infant / Male Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2018 Tipo del documento: Article
Texto completo
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Terapia Genética / Adenosina Desaminasa / Inmunodeficiencia Combinada Grave / Agammaglobulinemia Tipo de estudio: Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Child / Child, preschool / Humans / Infant / Male Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2018 Tipo del documento: Article