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Base editing repairs an SGCA mutation in human primary muscle stem cells.
Escobar, Helena; Krause, Anne; Keiper, Sandra; Kieshauer, Janine; Müthel, Stefanie; de Paredes, Manuel García; Metzler, Eric; Kühn, Ralf; Heyd, Florian; Spuler, Simone.
Afiliação
  • Escobar H; Muscle Research Unit, Experimental and Clinical Research Center, a cooperation between the Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association and the Charité, Universitätsmedizin Berlin, Germany.
  • Krause A; Charité Universitätsmedizin Berlin, Germany.
  • Keiper S; Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association, Berlin, Germany.
  • Kieshauer J; Muscle Research Unit, Experimental and Clinical Research Center, a cooperation between the Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association and the Charité, Universitätsmedizin Berlin, Germany.
  • Müthel S; Charité Universitätsmedizin Berlin, Germany.
  • de Paredes MG; Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association, Berlin, Germany.
  • Metzler E; Freie Universität Berlin, Institute of Chemistry and Biochemistry, Laboratory of RNA Biochemistry, Berlin, Germany.
  • Kühn R; Muscle Research Unit, Experimental and Clinical Research Center, a cooperation between the Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association and the Charité, Universitätsmedizin Berlin, Germany.
  • Heyd F; Charité Universitätsmedizin Berlin, Germany.
  • Spuler S; Max-Delbrück-Center for Molecular Medicine in the Helmholtz Association, Berlin, Germany.
JCI Insight ; 6(10)2021 05 24.
Article em En | MEDLINE | ID: mdl-33848270
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Músculo Esquelético / Mioblastos / Sarcoglicanas / Edição de Genes / Mutação Limite: Adolescent / Animals / Child / Female / Humans / Male Idioma: En Ano de publicação: 2021 Tipo de documento: Article
Texto completo
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PubMed Links
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Músculo Esquelético / Mioblastos / Sarcoglicanas / Edição de Genes / Mutação Limite: Adolescent / Animals / Child / Female / Humans / Male Idioma: En Ano de publicação: 2021 Tipo de documento: Article