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CRISPR-Cas9 homology-independent targeted integration of exons 1-19 restores full-length dystrophin in mice.
Stephenson, Anthony A; Nicolau, Stefan; Vetter, Tatyana A; Dufresne, Gabrielle P; Frair, Emma C; Sarff, Jessica E; Wheeler, Gregory L; Kelly, Benjamin J; White, Peter; Flanigan, Kevin M.
Afiliación
  • Stephenson AA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • Nicolau S; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • Vetter TA; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • Dufresne GP; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA.
  • Frair EC; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • Sarff JE; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • Wheeler GL; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • Kelly BJ; The Institute for Genomic Medicine, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • White P; The Institute for Genomic Medicine, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • Flanigan KM; Department of Pediatrics, The Ohio State University, Columbus, OH 43210, USA.
Mol Ther Methods Clin Dev ; 30: 486-499, 2023 Sep 14.
Article en En | MEDLINE | ID: mdl-37706184
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos