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In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies.
Li, Chang; Georgakopoulou, Aphrodite; Newby, Gregory A; Everette, Kelcee A; Nizamis, Evangelos; Paschoudi, Kiriaki; Vlachaki, Efthymia; Gil, Sucheol; Anderson, Anna K; Koob, Theodore; Huang, Lishan; Wang, Hongjie; Kiem, Hans-Peter; Liu, David R; Yannaki, Evangelia; Lieber, André.
Afiliação
  • Li C; Department of Medicine, Division of Medical Genetics, University of Washington, Seattle, Washington, USA.
  • Georgakopoulou A; Gene and Cell Therapy Center, Hematology Department, George Papanicolaou Hospital, Thessaloniki, Greece.
  • Newby GA; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, Massachusetts, USA.
  • Everette KA; Department of Chemistry and Chemical Biology and.
  • Nizamis E; Howard Hughes Medical Institute, Harvard University, Cambridge, Massachusetts, USA.
  • Paschoudi K; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, Massachusetts, USA.
  • Vlachaki E; Department of Chemistry and Chemical Biology and.
  • Gil S; Howard Hughes Medical Institute, Harvard University, Cambridge, Massachusetts, USA.
  • Anderson AK; Department of Computer Science and Biomedical Informatics, University of Thessaly, Lamia, Greece.
  • Koob T; Gene and Cell Therapy Center, Hematology Department, George Papanicolaou Hospital, Thessaloniki, Greece.
  • Huang L; School of Biology, Aristotle University of Thessaloniki, Thessaloniki, Greece.
  • Wang H; Hematological Laboratory, Second Department of Internal Medicine, Faculty of Health Sciences, School of Medicine, Aristotle University of Thessaloniki, Hippokration General Hospital, Thessaloniki, Greece.
  • Kiem HP; Department of Medicine, Division of Medical Genetics, University of Washington, Seattle, Washington, USA.
  • Liu DR; Department of Medicine, Division of Medical Genetics, University of Washington, Seattle, Washington, USA.
  • Yannaki E; Department of Medicine, Division of Medical Genetics, University of Washington, Seattle, Washington, USA.
  • Lieber A; Department of Medicine, Division of Medical Genetics, University of Washington, Seattle, Washington, USA.
JCI Insight ; 7(19)2022 10 10.
Article em En | MEDLINE | ID: mdl-36006707
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Talassemia beta / Hemoglobinopatias / Anemia Falciforme Limite: Animals / Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Talassemia beta / Hemoglobinopatias / Anemia Falciforme Limite: Animals / Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article