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AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD.
Giesige, Carlee R; Wallace, Lindsay M; Heller, Kristin N; Eidahl, Jocelyn O; Saad, Nizar Y; Fowler, Allison M; Pyne, Nettie K; Al-Kharsan, Mustafa; Rashnonejad, Afrooz; Chermahini, Gholamhossein Amini; Domire, Jacqueline S; Mukweyi, Diana; Garwick-Coppens, Sara E; Guckes, Susan M; McLaughlin, K John; Meyer, Kathrin; Rodino-Klapac, Louise R; Harper, Scott Q.
Afiliación
  • Giesige CR; Biomedical Sciences Graduate Program, The Ohio State University, Columbus, Ohio, USA.
  • Wallace LM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Heller KN; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Eidahl JO; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Saad NY; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Fowler AM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Pyne NK; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Al-Kharsan M; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Rashnonejad A; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Chermahini GA; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Domire JS; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Mukweyi D; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Garwick-Coppens SE; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Guckes SM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • McLaughlin KJ; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Meyer K; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
  • Rodino-Klapac LR; Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
  • Harper SQ; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.
JCI Insight ; 3(22)2018 11 15.
Article en En | MEDLINE | ID: mdl-30429376
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Proteínas de Homeodominio / Distrofia Muscular Facioescapulohumeral / Folistatina / Modelos Animales de Enfermedad / Miostatina Tipo de estudio: Prognostic_studies Límite: Animals Idioma: En Revista: JCI Insight Año: 2018 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Proteínas de Homeodominio / Distrofia Muscular Facioescapulohumeral / Folistatina / Modelos Animales de Enfermedad / Miostatina Tipo de estudio: Prognostic_studies Límite: Animals Idioma: En Revista: JCI Insight Año: 2018 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos