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Base editing rescue of spinal muscular atrophy in cells and in mice.
Arbab, Mandana; Matuszek, Zaneta; Kray, Kaitlyn M; Du, Ailing; Newby, Gregory A; Blatnik, Anton J; Raguram, Aditya; Richter, Michelle F; Zhao, Kevin T; Levy, Jonathan M; Shen, Max W; Arnold, W David; Wang, Dan; Xie, Jun; Gao, Guangping; Burghes, Arthur H M; Liu, David R.
Afiliação
  • Arbab M; Department of Neurology, Rosamund Stone Zander Translational Neuroscience Center, Boston Children's Hospital, Boston, MA 02115, USA.
  • Matuszek Z; Department of Neurobiology, Harvard Medical School, Boston, MA 02115, USA.
  • Kray KM; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
  • Du A; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA 02138, USA.
  • Newby GA; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
  • Blatnik AJ; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA 02138, USA.
  • Raguram A; Department of Molecular and Cellular Biology, Harvard University, Cambridge, MA 02138, USA.
  • Richter MF; Department of Biological Chemistry and Pharmacology, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA.
  • Zhao KT; Horae Gene Therapy Center, UMass Chan Medical School, University of Massachusetts, Worcester, MA 01605, USA.
  • Levy JM; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
  • Shen MW; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA 02138, USA.
  • Arnold WD; Department of Biological Chemistry and Pharmacology, The Ohio State University Wexner Medical Center, Columbus, OH 43210, USA.
  • Wang D; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
  • Xie J; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA 02138, USA.
  • Gao G; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
  • Burghes AHM; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA 02138, USA.
  • Liu DR; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
Science ; 380(6642): eadg6518, 2023 04 21.
Article em En | MEDLINE | ID: mdl-36996170
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal / Proteína 1 de Sobrevivência do Neurônio Motor / Proteína 2 de Sobrevivência do Neurônio Motor / Edição de Genes Limite: Animals Idioma: En Revista: Science Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal / Proteína 1 de Sobrevivência do Neurônio Motor / Proteína 2 de Sobrevivência do Neurônio Motor / Edição de Genes Limite: Animals Idioma: En Revista: Science Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos