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Base editing of haematopoietic stem cells rescues sickle cell disease in mice.
Newby, Gregory A; Yen, Jonathan S; Woodard, Kaitly J; Mayuranathan, Thiyagaraj; Lazzarotto, Cicera R; Li, Yichao; Sheppard-Tillman, Heather; Porter, Shaina N; Yao, Yu; Mayberry, Kalin; Everette, Kelcee A; Jang, Yoonjeong; Podracky, Christopher J; Thaman, Elizabeth; Lechauve, Christophe; Sharma, Akshay; Henderson, Jordana M; Richter, Michelle F; Zhao, Kevin T; Miller, Shannon M; Wang, Tina; Koblan, Luke W; McCaffrey, Anton P; Tisdale, John F; Kalfa, Theodosia A; Pruett-Miller, Shondra M; Tsai, Shengdar Q; Weiss, Mitchell J; Liu, David R.
Afiliação
  • Newby GA; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
  • Yen JS; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.
  • Woodard KJ; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA.
  • Mayuranathan T; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA. jonathan.yen@stjude.org.
  • 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.
  • Sheppard-Tillman H; 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.
  • Yao Y; Department of Pathology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Mayberry K; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Everette KA; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Jang Y; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Podracky CJ; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
  • Thaman E; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.
  • Lechauve C; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA.
  • Sharma A; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Henderson JM; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
  • Richter MF; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.
  • Zhao KT; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA.
  • Miller SM; Division of Hematology, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
  • Wang T; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Koblan LW; Department of Bone Marrow Transplantation and Cellular Therapy, St Jude Children's Research Hospital, Memphis, TN, USA.
  • McCaffrey AP; TriLink BioTechnologies, San Diego, CA, USA.
  • Tisdale JF; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
  • Kalfa TA; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.
  • Pruett-Miller SM; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA.
  • Tsai SQ; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
  • Weiss MJ; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.
  • Liu DR; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA.
Nature ; 595(7866): 295-302, 2021 07.
Article em En | MEDLINE | ID: mdl-34079130
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Células-Tronco Hematopoéticas / Adenina / Transplante de Células-Tronco Hematopoéticas / Globinas beta / Edição de Genes / Anemia Falciforme Tipo de estudo: Prognostic_studies Limite: Animals / Female / Humans / Male Idioma: En Revista: Nature Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Células-Tronco Hematopoéticas / Adenina / Transplante de Células-Tronco Hematopoéticas / Globinas beta / Edição de Genes / Anemia Falciforme Tipo de estudo: Prognostic_studies Limite: Animals / Female / Humans / Male Idioma: En Revista: Nature Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos