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Natural gene therapy by reverse mosaicism leads to improved hematology in Fanconi anemia patients.
Ramírez, María José; Pujol, Roser; Trujillo-Quintero, Juan Pablo; Minguillón, Jordi; Bogliolo, Massimo; Río, Paula; Navarro, Susana; Casado, José A; Badell, Isabel; Carrasco, Estela; Balmaña, Judith; Català, Albert; Sevilla, Julián; Beléndez, Cristina; Argilés, Bienvenida; López, Mónica; Díaz de Heredia, Cristina; Rao, Gayatri; Nicoletti, Eileen; Schwartz, Jonathan D; Bueren, Juan A; Surrallés, Jordi.
Afiliación
  • Ramírez MJ; Genomic Instability and DNA Repair Syndromes Group and Joint Research Unit on Genomic Medicine UAB-Sant Pau Biomedical Research Institute (IIB Sant Pau), Institut de Recerca Hospital de la Santa Creu i Sant Pau-IIB Sant Pau, Barcelona, Spain.
  • Pujol R; Department of Genetics and Microbiology, Universitat Autònoma de Barcelona, Barcelona, Spain.
  • Trujillo-Quintero JP; Center for Biomedical Network Research on Rare Diseases (CIBERER), Madrid, Spain.
  • Minguillón J; Genomic Instability and DNA Repair Syndromes Group and Joint Research Unit on Genomic Medicine UAB-Sant Pau Biomedical Research Institute (IIB Sant Pau), Institut de Recerca Hospital de la Santa Creu i Sant Pau-IIB Sant Pau, Barcelona, Spain.
  • Bogliolo M; Department of Genetics and Microbiology, Universitat Autònoma de Barcelona, Barcelona, Spain.
  • Río P; Center for Biomedical Network Research on Rare Diseases (CIBERER), Madrid, Spain.
  • Navarro S; Department of Genetics and Microbiology, Universitat Autònoma de Barcelona, Barcelona, Spain.
  • Casado JA; Unitat de Genètica Clínica Pediàtrica, Parc Taulí Hospital Universitari, Barcelona, Spain.
  • Badell I; Genomic Instability and DNA Repair Syndromes Group and Joint Research Unit on Genomic Medicine UAB-Sant Pau Biomedical Research Institute (IIB Sant Pau), Institut de Recerca Hospital de la Santa Creu i Sant Pau-IIB Sant Pau, Barcelona, Spain.
  • Carrasco E; Department of Genetics and Microbiology, Universitat Autònoma de Barcelona, Barcelona, Spain.
  • Balmaña J; Center for Biomedical Network Research on Rare Diseases (CIBERER), Madrid, Spain.
  • Català A; Genomic Instability and DNA Repair Syndromes Group and Joint Research Unit on Genomic Medicine UAB-Sant Pau Biomedical Research Institute (IIB Sant Pau), Institut de Recerca Hospital de la Santa Creu i Sant Pau-IIB Sant Pau, Barcelona, Spain.
  • Sevilla J; Department of Genetics and Microbiology, Universitat Autònoma de Barcelona, Barcelona, Spain.
  • Beléndez C; Center for Biomedical Network Research on Rare Diseases (CIBERER), Madrid, Spain.
  • Argilés B; Center for Biomedical Network Research on Rare Diseases (CIBERER), Madrid, Spain.
  • López M; Division of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
  • Díaz de Heredia C; Advanced Therapies Unit, IIS-Fundacion Jimenez Diaz (IIS-FJD, UAM), Madrid, Spain.
  • Rao G; Center for Biomedical Network Research on Rare Diseases (CIBERER), Madrid, Spain.
  • Nicoletti E; Division of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
  • Schwartz JD; Advanced Therapies Unit, IIS-Fundacion Jimenez Diaz (IIS-FJD, UAM), Madrid, Spain.
  • Bueren JA; Center for Biomedical Network Research on Rare Diseases (CIBERER), Madrid, Spain.
  • Surrallés J; Division of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Madrid, Spain.
Am J Hematol ; 96(8): 989-999, 2021 08 01.
Article en En | MEDLINE | ID: mdl-33984160
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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Terapia Genética / Anemia de Fanconi Tipo de estudio: Prognostic_studies Límite: Adolescent / Adult / Child / Humans / Male Idioma: En Revista: Am J Hematol Año: 2021 Tipo del documento: Article País de afiliación: España
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Terapia Genética / Anemia de Fanconi Tipo de estudio: Prognostic_studies Límite: Adolescent / Adult / Child / Humans / Male Idioma: En Revista: Am J Hematol Año: 2021 Tipo del documento: Article País de afiliación: España