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Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy.
Reilly, Aoife; Deguise, Marc-Olivier; Beauvais, Ariane; Yaworski, Rebecca; Thebault, Simon; Tessier, Daniel R; Tabard-Cossa, Vincent; Hensel, Niko; Schneider, Bernard L; Kothary, Rashmi.
Afiliación
  • Reilly A; Regenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, Canada.
  • Deguise MO; Department of Cellular and Molecular Medicine, University of Ottawa, Ottawa, Canada.
  • Beauvais A; Centre for Neuromuscular Disease, University of Ottawa, Ottawa, Canada.
  • Yaworski R; Regenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, Canada.
  • Thebault S; Department of Cellular and Molecular Medicine, University of Ottawa, Ottawa, Canada.
  • Tessier DR; Centre for Neuromuscular Disease, University of Ottawa, Ottawa, Canada.
  • Tabard-Cossa V; Regenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, Canada.
  • Hensel N; Regenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, Canada.
  • Schneider BL; Division of Neurology, Department of Medicine, The Ottawa Hospital, Ottawa, Canada.
  • Kothary R; Department of Medicine, University of Ottawa, Ottawa, Canada.
Gene Ther ; 29(9): 544-554, 2022 09.
Article en En | MEDLINE | ID: mdl-35462564

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Atrofia Muscular Espinal / Dependovirus Límite: Animals Idioma: En Revista: Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2022 Tipo del documento: Article País de afiliación: Canadá

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Atrofia Muscular Espinal / Dependovirus Límite: Animals Idioma: En Revista: Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2022 Tipo del documento: Article País de afiliación: Canadá