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A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease.
Cai, Liuhong; Bai, Hao; Mahairaki, Vasiliki; Gao, Yongxing; He, Chaoxia; Wen, Yanfei; Jin, You-Chuan; Wang, You; Pan, Rachel L; Qasba, Armaan; Ye, Zhaohui; Cheng, Linzhao.
Afiliação
  • Cai L; Center for Reproductive Medicine, The Third Affiliated Hospital, Sun Yat-sen University, Guangzhou, People's Republic of China.
  • Bai H; Division of Hematology, Department of Medicine, Baltimore, Maryland, USA.
  • Mahairaki V; Stem Cell Program in the Institute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Gao Y; Division of Hematology, Department of Medicine, Baltimore, Maryland, USA.
  • He C; Stem Cell Program in the Institute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Wen Y; Stem Cell Program in the Institute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Jin YC; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Wang Y; Division of Hematology, Department of Medicine, Baltimore, Maryland, USA.
  • Pan RL; Stem Cell Program in the Institute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Qasba A; Division of Hematology, Department of Medicine, Baltimore, Maryland, USA.
  • Ye Z; Stem Cell Program in the Institute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Cheng L; Center for Reproductive Medicine, The Third Affiliated Hospital, Sun Yat-sen University, Guangzhou, People's Republic of China.
Stem Cells Transl Med ; 7(1): 87-97, 2018 01.
Article em En | MEDLINE | ID: mdl-29164808

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Talassemia beta / Globinas beta / Células-Tronco Pluripotentes Induzidas / Edição de Genes / Anemia Falciforme Limite: Female / Humans / Male Idioma: En Revista: Stem Cells Transl Med Ano de publicação: 2018 Tipo de documento: Article País de publicação: Reino Unido

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Talassemia beta / Globinas beta / Células-Tronco Pluripotentes Induzidas / Edição de Genes / Anemia Falciforme Limite: Female / Humans / Male Idioma: En Revista: Stem Cells Transl Med Ano de publicação: 2018 Tipo de documento: Article País de publicação: Reino Unido