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Correction of IVS I-110(G>A) ß-thalassemia by CRISPR/Cas-and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells.
Patsali, Petros; Turchiano, Giandomenico; Papasavva, Panayiota; Romito, Marianna; Loucari, Constantinos C; Stephanou, Coralea; Christou, Soteroulla; Sitarou, Maria; Mussolino, Claudio; Cornu, Tatjana I; Antoniou, Michael N; Lederer, Carsten W; Cathomen, Toni; Kleanthous, Marina.
Afiliação
  • Patsali P; Department of Molecular Genetics Thalassemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
  • Turchiano G; Department of Medical and Molecular Genetics, King's College London, London, UK.
  • Papasavva P; Institute for Transfusion Medicine and Gene Therapy, Medical Center, University of Freiburg, Freiburg, Germany.
  • Romito M; Center for Chronic Immunodeficiency, Medical Center, University of Freiburg, Freiburg, Germany.
  • Loucari CC; Department of Molecular Genetics Thalassemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
  • Stephanou C; Cyprus School of Molecular Medicine, Nicosia, Cyprus.
  • Christou S; Institute for Transfusion Medicine and Gene Therapy, Medical Center, University of Freiburg, Freiburg, Germany.
  • Sitarou M; Center for Chronic Immunodeficiency, Medical Center, University of Freiburg, Freiburg, Germany.
  • Mussolino C; Department of Molecular Genetics Thalassemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
  • Cornu TI; Cyprus School of Molecular Medicine, Nicosia, Cyprus.
  • Antoniou MN; Department of Molecular Genetics Thalassemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
  • Lederer CW; Department of Medical and Molecular Genetics, King's College London, London, UK.
  • Cathomen T; Thalassemia Center, Cyprus Ministry of Health, Cyprus.
  • Kleanthous M; Thalassemia Center, Cyprus Ministry of Health, Cyprus.
Haematologica ; 104(11): e497-e501, 2019 11.
Article em En | MEDLINE | ID: mdl-31004018
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Células-Tronco Hematopoéticas / Íntrons / Talassemia beta / Globinas beta / Sistemas CRISPR-Cas / Nucleases dos Efetores Semelhantes a Ativadores de Transcrição / Edição de Genes Limite: Humans Idioma: En Revista: Haematologica Ano de publicação: 2019 Tipo de documento: Article
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Células-Tronco Hematopoéticas / Íntrons / Talassemia beta / Globinas beta / Sistemas CRISPR-Cas / Nucleases dos Efetores Semelhantes a Ativadores de Transcrição / Edição de Genes Limite: Humans Idioma: En Revista: Haematologica Ano de publicação: 2019 Tipo de documento: Article