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Potent and uniform fetal hemoglobin induction via base editing.
Mayuranathan, Thiyagaraj; Newby, Gregory A; Feng, Ruopeng; Yao, Yu; Mayberry, Kalin D; Lazzarotto, Cicera R; Li, Yichao; Levine, Rachel M; Nimmagadda, Nikitha; Dempsey, Erin; Kang, Guolian; Porter, Shaina N; Doerfler, Phillip A; Zhang, Jingjing; Jang, Yoonjeong; Chen, Jingjing; Bell, Henry W; Crossley, Merlin; Bhoopalan, Senthil Velan; Sharma, Akshay; Tisdale, John F; Pruett-Miller, Shondra M; Cheng, Yong; Tsai, Shengdar Q; Liu, David R; Weiss, Mitchell J; Yen, Jonathan S.
Afiliação
  • Mayuranathan T; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Newby GA; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
  • Feng R; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.
  • Yao Y; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA.
  • Mayberry KD; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Lazzarotto CR; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Li Y; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Levine RM; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Nimmagadda N; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Dempsey E; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Kang G; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Porter SN; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Doerfler PA; Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Zhang J; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Jang Y; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Chen J; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Bell HW; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Crossley M; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Bhoopalan SV; School of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney, New South Wales, Australia.
  • Sharma A; School of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney, New South Wales, Australia.
  • Tisdale JF; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Pruett-Miller SM; Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Cheng Y; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute and National Institute of Diabetes and Digestive and Kidney Diseases, Bethesda, MD, USA.
  • Tsai SQ; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Liu DR; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Weiss MJ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Yen JS; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA. drliu@fas.harvard.edu.
Nat Genet ; 55(7): 1210-1220, 2023 07.
Article em En | MEDLINE | ID: mdl-37400614

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Talassemia beta / Anemia Falciforme Limite: Animals Idioma: En Ano de publicação: 2023 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Talassemia beta / Anemia Falciforme Limite: Animals Idioma: En Ano de publicação: 2023 Tipo de documento: Article