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Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse.
Wein, Nicolas; Vetter, Tatyana A; Vulin, Adeline; Simmons, Tabatha R; Frair, Emma C; Bradley, Adrienne J; Gushchina, Liubov V; Almeida, Camila F; Huang, Nianyuan; Lesman, Daniel; Rajakumar, Dhanarajan; Weiss, Robert B; Flanigan, Kevin M.
Affiliation
  • Wein N; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
  • Vetter TA; Department of Pediatrics, The Ohio State University, Columbus, OH, USA.
  • Vulin A; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
  • Simmons TR; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
  • Frair EC; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
  • Bradley AJ; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
  • Gushchina LV; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
  • Almeida CF; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
  • Huang N; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
  • Lesman D; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
  • Rajakumar D; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
  • Weiss RB; Center for Gene Therapy, The Abigail Wexner Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, OH 43205, USA.
  • Flanigan KM; Department of Human Genetics, The University of Utah School of Medicine, Salt Lake City, UT, USA.
Mol Ther Methods Clin Dev ; 26: 279-293, 2022 Sep 08.
Article de En | MEDLINE | ID: mdl-35949298
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Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Langue: En Journal: Mol Ther Methods Clin Dev Année: 2022 Type de document: Article Pays d'affiliation: États-Unis d'Amérique Pays de publication: États-Unis d'Amérique
Texte intégral
Ajouter à My VHL
Imprimer
XML
PubMed Links
Recherche sur Google

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