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Curative therapy for hemoglobinopathies: an International Society for Cell & Gene Therapy Stem Cell Engineering Committee review comparing outcomes, accessibility and cost of ex vivo stem cell gene therapy versus allogeneic hematopoietic stem cell transplantation.
Leonard, Alexis; Bertaina, Alice; Bonfim, Carmem; Cohen, Sandra; Prockop, Susan; Purtill, Duncan; Russell, Athena; Boelens, Jaap Jan; Wynn, Robert; Ruggeri, Annalisa; Abraham, Allistair.
Afiliación
  • Leonard A; Division of Hematology, Children's National Hospital, Washington, DC, USA.
  • Bertaina A; Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University, Stanford, California, USA.
  • Bonfim C; Pediatric Bone Marrow Transplantation Division, Hospital Pequeno Principe, Curitiba, Brazil.
  • Cohen S; Université de Montréal and Maisonneuve Rosemont Hospital, Montréal, Canada.
  • Prockop S; Stem Cell Transplantation and Cellular Therapies, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, New York, USA.
  • Purtill D; Department of Haematology, Fiona Stanley Hospital, Perth, Australia.
  • Russell A; Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Boelens JJ; Stem Cell Transplantation and Cellular Therapies, Memorial Sloan Kettering Cancer Center, New York, New York, USA; Department of Pediatrics, Weill Cornell Medical College of Cornell University, New York, New York, USA.
  • Wynn R; Faculty of Biology, Medicine and Health, The University of Manchester, Manchester, UK.
  • Ruggeri A; Department of Hematology and bone marrow transplantation, IRCCS Ospedale San Raffaele, Segrate, Milan, Italy.
  • Abraham A; Center for Cancer and Immunology Research, CETI, Children's National Hospital, Washington, DC, USA. Electronic address: aabraham@childrensnational.org.
Cytotherapy ; 24(3): 249-261, 2022 03.
Article en En | MEDLINE | ID: mdl-34879990
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Texto completo: 1 Colección: 01-internacional Asunto principal: Talasemia beta / Trasplante de Células Madre Hematopoyéticas / Enfermedad Injerto contra Huésped / Hemoglobinopatías / Anemia de Células Falciformes Tipo de estudio: Health_economic_evaluation Límite: Humans Idioma: En Revista: Cytotherapy Asunto de la revista: TERAPEUTICA Año: 2022 Tipo del documento: Article País de afiliación: Estados Unidos
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Texto completo: 1 Colección: 01-internacional Asunto principal: Talasemia beta / Trasplante de Células Madre Hematopoyéticas / Enfermedad Injerto contra Huésped / Hemoglobinopatías / Anemia de Células Falciformes Tipo de estudio: Health_economic_evaluation Límite: Humans Idioma: En Revista: Cytotherapy Asunto de la revista: TERAPEUTICA Año: 2022 Tipo del documento: Article País de afiliación: Estados Unidos