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Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice.
Lahey, Hannah G; Webber, Chelsea J; Golebiowski, Diane; Izzo, Cassandra M; Horn, Erin; Taghian, Toloo; Rodriguez, Paola; Batista, Ana Rita; Ellis, Lauren E; Hwang, Misako; Martin, Douglas R; Gray-Edwards, Heather; Sena-Esteves, Miguel.
Afiliación
  • Lahey HG; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
  • Webber CJ; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
  • Golebiowski D; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
  • Izzo CM; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
  • Horn E; Department of Radiology, University of Massachusetts Medical School, Worcester, MA, USA; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
  • Taghian T; Department of Radiology, University of Massachusetts Medical School, Worcester, MA, USA; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
  • Rodriguez P; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
  • Batista AR; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
  • Ellis LE; Scott-Ritchey Research Center, Auburn University College of Veterinary Medicine, Auburn, AL, USA.
  • Hwang M; Scott-Ritchey Research Center, Auburn University College of Veterinary Medicine, Auburn, AL, USA.
  • Martin DR; Scott-Ritchey Research Center, Auburn University College of Veterinary Medicine, Auburn, AL, USA; Department of Anatomy, Physiology & Pharmacology, Auburn University College of Veterinary Medicine, Auburn, AL, USA.
  • Gray-Edwards H; Department of Radiology, University of Massachusetts Medical School, Worcester, MA, USA; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
  • Sena-Esteves M; Department of Neurology, University of Massachusetts Medical School, Worcester, MA, USA; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA. Electronic address: miguel.esteves@umassmed.edu.
Mol Ther ; 28(10): 2150-2160, 2020 10 07.
Article en En | MEDLINE | ID: mdl-32592687
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Contexto en salud: 6_ODS3_enfermedades_notrasmisibles Problema de salud: 6_endocrine_disorders Asunto principal: Enfermedad de Sandhoff / Terapia Genética / Dependovirus / Vectores Genéticos Límite: Animals Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2020 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Contexto en salud: 6_ODS3_enfermedades_notrasmisibles Problema de salud: 6_endocrine_disorders Asunto principal: Enfermedad de Sandhoff / Terapia Genética / Dependovirus / Vectores Genéticos Límite: Animals Idioma: En Revista: Mol Ther Asunto de la revista: BIOLOGIA MOLECULAR / TERAPEUTICA Año: 2020 Tipo del documento: Article País de afiliación: Estados Unidos