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Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy.
Potter, Rachael A; Griffin, Danielle A; Sondergaard, Patricia C; Johnson, Ryan W; Pozsgai, Eric R; Heller, Kristin N; Peterson, Ellyn L; Lehtimäki, Kimmo K; Windish, Hillarie P; Mittal, Plavi J; Albrecht, Douglas E; Mendell, Jerry R; Rodino-Klapac, Louise R.
Afiliación
  • Potter RA; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.
  • Griffin DA; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.
  • Sondergaard PC; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.
  • Johnson RW; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.
  • Pozsgai ER; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.
  • Heller KN; 3 Integrated Biomedical Science Graduate Program, College of Medicine, The Ohio State University , Columbus, Ohio; The Ohio State University, Columbus, Ohio.
  • Peterson EL; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.
  • Lehtimäki KK; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.
  • Windish HP; 5 Charles River Discovery Services , Kuopio, Finland .
  • Mittal PJ; 4 The Jain Foundation , Seattle, Washington.
  • Albrecht DE; 4 The Jain Foundation , Seattle, Washington.
  • Mendell JR; 4 The Jain Foundation , Seattle, Washington.
  • Rodino-Klapac LR; 1 Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital , Columbus, Ohio.
Hum Gene Ther ; 29(7): 749-762, 2018 07.
Article en En | MEDLINE | ID: mdl-28707952
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / ADN Complementario / Distrofia Muscular de Cinturas / Disferlina Tipo de estudio: Clinical_trials Límite: Animals / Humans / Male Idioma: En Revista: Hum Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2018 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
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XML
PubMed Links
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / ADN Complementario / Distrofia Muscular de Cinturas / Disferlina Tipo de estudio: Clinical_trials Límite: Animals / Humans / Male Idioma: En Revista: Hum Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2018 Tipo del documento: Article Pais de publicación: Estados Unidos