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Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy.
Gushchina, Liubov V; Vetter, Tatyana A; Frair, Emma C; Bradley, Adrienne J; Grounds, Kelly M; Lay, Jacob W; Huang, Nianyuan; Suhaiba, Aisha; Schnell, Frederick J; Hanson, Gunnar; Simmons, Tabatha R; Wein, Nicolas; Flanigan, Kevin M.
Affiliation
  • Gushchina LV; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
  • Vetter TA; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
  • Frair EC; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
  • Bradley AJ; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
  • Grounds KM; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
  • Lay JW; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
  • Huang N; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
  • Suhaiba A; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
  • Schnell FJ; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Hanson G; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Simmons TR; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
  • Wein N; The Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
  • Flanigan KM; Department of Pediatrics, The Ohio State University, Columbus, OH, USA.
Mol Ther Nucleic Acids ; 30: 479-492, 2022 Dec 13.
Article de En | MEDLINE | ID: mdl-36420217

Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Type d'étude: Prognostic_studies Langue: En Journal: Mol Ther Nucleic Acids Année: 2022 Type de document: Article Pays d'affiliation: États-Unis d'Amérique Pays de publication: États-Unis d'Amérique

Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Type d'étude: Prognostic_studies Langue: En Journal: Mol Ther Nucleic Acids Année: 2022 Type de document: Article Pays d'affiliation: États-Unis d'Amérique Pays de publication: États-Unis d'Amérique