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Correction of ß-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells.
Pavani, Giulia; Fabiano, Anna; Laurent, Marine; Amor, Fatima; Cantelli, Erika; Chalumeau, Anne; Maule, Giulia; Tachtsidi, Alexandra; Concordet, Jean-Paul; Cereseto, Anna; Mavilio, Fulvio; Ferrari, Giuliana; Miccio, Annarita; Amendola, Mario.
Afiliación
  • Pavani G; Integrated Genetic Approaches in Therapeutic Discovery for Rare Diseases (INTEGRARE), Genethon, Unité Mixte de Recherche (UMR) S951 INSERM, University Evry, University Paris-Saclay, Evry, France.
  • Fabiano A; Integrated Genetic Approaches in Therapeutic Discovery for Rare Diseases (INTEGRARE), Genethon, Unité Mixte de Recherche (UMR) S951 INSERM, University Evry, University Paris-Saclay, Evry, France.
  • Laurent M; Integrated Genetic Approaches in Therapeutic Discovery for Rare Diseases (INTEGRARE), Genethon, Unité Mixte de Recherche (UMR) S951 INSERM, University Evry, University Paris-Saclay, Evry, France.
  • Amor F; Integrated Genetic Approaches in Therapeutic Discovery for Rare Diseases (INTEGRARE), Genethon, Unité Mixte de Recherche (UMR) S951 INSERM, University Evry, University Paris-Saclay, Evry, France.
  • Cantelli E; Integrated Genetic Approaches in Therapeutic Discovery for Rare Diseases (INTEGRARE), Genethon, Unité Mixte de Recherche (UMR) S951 INSERM, University Evry, University Paris-Saclay, Evry, France.
  • Chalumeau A; Imagine Institute, UMR 163 INSERM, Paris, France.
  • Maule G; Paris Descartes, University Sorbonne Paris Cité, Paris, France.
  • Tachtsidi A; Centre for Integrative Biology (CIBIO), University of Trento, Trento, Italy.
  • Concordet JP; Institute of Biophysics, National Research Council, Trento, Italy.
  • Cereseto A; Integrated Genetic Approaches in Therapeutic Discovery for Rare Diseases (INTEGRARE), Genethon, Unité Mixte de Recherche (UMR) S951 INSERM, University Evry, University Paris-Saclay, Evry, France.
  • Mavilio F; National Museum of Natural History, UMR_1154 INSERM, UMR_7196 CNRS, University Sorbonne, Paris, France.
  • Ferrari G; Centre for Integrative Biology (CIBIO), University of Trento, Trento, Italy.
  • Miccio A; Department of Life Sciences, University of Modena and Reggio Emilia, Modena, Italy; and.
  • Amendola M; Vita-Salute San Raffaele University, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Blood Adv ; 5(5): 1137-1153, 2021 03 09.
Article en En | MEDLINE | ID: mdl-33635334
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Talasemia beta Límite: Animals / Humans Idioma: En Revista: Blood Adv Año: 2021 Tipo del documento: Article País de afiliación: Francia
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Talasemia beta Límite: Animals / Humans Idioma: En Revista: Blood Adv Año: 2021 Tipo del documento: Article País de afiliación: Francia