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Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice.
Everette, Kelcee A; Newby, Gregory A; Levine, Rachel M; Mayberry, Kalin; Jang, Yoonjeong; Mayuranathan, Thiyagaraj; Nimmagadda, Nikitha; Dempsey, Erin; Li, Yichao; Bhoopalan, Senthil Velan; Liu, Xiong; Davis, Jessie R; Nelson, Andrew T; Chen, Peter J; Sousa, Alexander A; Cheng, Yong; Tisdale, John F; Weiss, Mitchell J; Yen, Jonathan S; Liu, David R.
Afiliação
  • Everette KA; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
  • Newby GA; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.
  • Levine RM; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA.
  • Mayberry K; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
  • Jang Y; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.
  • Mayuranathan T; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, 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.
  • Li Y; Department of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.
  • Bhoopalan SV; Department of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.
  • Liu X; Department of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.
  • Davis JR; Department of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.
  • Nelson AT; Department of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.
  • Chen PJ; Department of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.
  • Sousa AA; Molecular and Clinical Hematology Branch, National Heart, Lung, and Blood Institute/National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Cheng Y; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
  • Tisdale JF; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.
  • Weiss MJ; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA.
  • Yen JS; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
  • Liu DR; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.
Nat Biomed Eng ; 7(5): 616-628, 2023 05.
Article em En | MEDLINE | ID: mdl-37069266
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Edição de Genes / Anemia Falciforme Tipo de estudo: Prognostic_studies Limite: Adult / Animals / Humans Idioma: En Revista: Nat Biomed Eng Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Edição de Genes / Anemia Falciforme Tipo de estudo: Prognostic_studies Limite: Adult / Animals / Humans Idioma: En Revista: Nat Biomed Eng Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos