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Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe's Disease after Global Gene Therapy.
Marshall, Michael S; Issa, Yazan; Jakubauskas, Benas; Stoskute, Monika; Elackattu, Vince; Marshall, Jeffrey N; Bogue, Wil; Nguyen, Duc; Hauck, Zane; Rue, Emily; Karumuthil-Melethil, Subha; Zaric, Violeta; Bosland, Maarten; van Breemen, Richard B; Givogri, Maria I; Gray, Steven J; Crocker, Stephen J; Bongarzone, Ernesto R.
Afiliación
  • Marshall MS; Department of Anatomy and Cell Biology, College of Medicine, University of Illinois at Chicago, Chicago, IL, USA.
  • Issa Y; Department of Anatomy and Cell Biology, College of Medicine, University of Illinois at Chicago, Chicago, IL, USA.
  • Jakubauskas B; Department of Anatomy and Cell Biology, College of Medicine, University of Illinois at Chicago, Chicago, IL, USA.
  • Stoskute M; Department of Anatomy and Cell Biology, College of Medicine, University of Illinois at Chicago, Chicago, IL, USA.
  • Elackattu V; Department of Anatomy and Cell Biology, College of Medicine, University of Illinois at Chicago, Chicago, IL, USA.
  • Marshall JN; Department of Anatomy and Cell Biology, College of Medicine, University of Illinois at Chicago, Chicago, IL, USA.
  • Bogue W; Department of Anatomy and Cell Biology, College of Medicine, University of Illinois at Chicago, Chicago, IL, USA.
  • Nguyen D; Department of Anatomy and Cell Biology, College of Medicine, University of Illinois at Chicago, Chicago, IL, USA.
  • Hauck Z; Department of Medicinal Chemistry and Pharmacognosy, College of Pharmacy, University of Illinois at Chicago, Chicago, IL, USA.
  • Rue E; Department of Medicinal Chemistry and Pharmacognosy, College of Pharmacy, University of Illinois at Chicago, Chicago, IL, USA.
  • Karumuthil-Melethil S; Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
  • Zaric V; Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
  • Bosland M; Department of Pathology, College of Medicine, University of Illinois at Chicago, Chicago, IL, USA.
  • van Breemen RB; Department of Medicinal Chemistry and Pharmacognosy, College of Pharmacy, University of Illinois at Chicago, Chicago, IL, USA.
  • Givogri MI; Department of Anatomy and Cell Biology, College of Medicine, University of Illinois at Chicago, Chicago, IL, USA.
  • Gray SJ; Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA; Department of Ophthalmology, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
  • Crocker SJ; Department of Neuroscience, University of Connecticut School of Medicine, Farmington, CT, USA.
  • Bongarzone ER; Department of Anatomy and Cell Biology, College of Medicine, University of Illinois at Chicago, Chicago, IL, USA; Departamento de Química Biológica, Facultad de Farmacia y Bioquímica, Universidad de Buenos Aires, 1053 Buenos Aires, Argentina. Electronic address: ebongarz@uic.edu.
Mol Ther ; 26(3): 874-889, 2018 03 07.
Article en En | MEDLINE | ID: mdl-29433937
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Fenotipo / Terapia Genética / Metabolismo de los Hidratos de Carbono / Galactosilceramidasa / Leucodistrofia de Células Globoides Tipo de estudio: Diagnostic_studies / Guideline / Prognostic_studies Límite: Animals Idioma: En Año: 2018 Tipo del documento: Article
Texto completo
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Fenotipo / Terapia Genética / Metabolismo de los Hidratos de Carbono / Galactosilceramidasa / Leucodistrofia de Células Globoides Tipo de estudio: Diagnostic_studies / Guideline / Prognostic_studies Límite: Animals Idioma: En Año: 2018 Tipo del documento: Article