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Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons.
Pearson, Toni S; Gupta, Nalin; San Sebastian, Waldy; Imamura-Ching, Jill; Viehoever, Amy; Grijalvo-Perez, Ana; Fay, Alex J; Seth, Neha; Lundy, Shannon M; Seo, Youngho; Pampaloni, Miguel; Hyland, Keith; Smith, Erin; de Oliveira Barbosa, Gardenia; Heathcock, Jill C; Minnema, Amy; Lonser, Russell; Elder, J Bradley; Leonard, Jeffrey; Larson, Paul; Bankiewicz, Krystof S.
  • Pearson TS; Department of Neurological Surgery, University of California San Francisco, San Francisco, CA, USA.
  • Gupta N; Department of Neurology, Washington University School of Medicine, St. Louis, MO, USA.
  • San Sebastian W; Department of Neurological Surgery, University of California San Francisco, San Francisco, CA, USA.
  • Imamura-Ching J; Department of Neurological Surgery, University of California San Francisco, San Francisco, CA, USA.
  • Viehoever A; Department of Neurological Surgery, University of California San Francisco, San Francisco, CA, USA.
  • Grijalvo-Perez A; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.
  • Fay AJ; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.
  • Seth N; Department of Neurology, University of California San Francisco, San Francisco, CA, USA.
  • Lundy SM; Department of Rehabilitative Services, University of California San Francisco, San Francisco, CA, USA.
  • Seo Y; Department of Pediatrics, University of California San Francisco, San Francisco, CA, USA.
  • Pampaloni M; Department of Radiology and Biomedical Imaging, University of California San Francisco, San Francisco, CA, USA.
  • Hyland K; Department of Radiology and Biomedical Imaging, University of California San Francisco, San Francisco, CA, USA.
  • Smith E; Medical Neurogenetics Laboratories, Atlanta, GA, USA.
  • de Oliveira Barbosa G; Therapy Services, St. Louis Children's Hospital, St. Louis, MO, USA.
  • Heathcock JC; School of Health and Rehabilitation Sciences, The Ohio State University, Columbus, OH, USA.
  • Minnema A; School of Health and Rehabilitation Sciences, The Ohio State University, Columbus, OH, USA.
  • Lonser R; Department of Neurological Surgery, The Ohio State University, Columbus, OH, USA.
  • Elder JB; Department of Neurological Surgery, The Ohio State University, Columbus, OH, USA.
  • Leonard J; Department of Neurological Surgery, The Ohio State University, Columbus, OH, USA.
  • Larson P; Department of Neurological Surgery, The Ohio State University, Columbus, OH, USA.
  • Bankiewicz KS; Department of Neurological Surgery, Nationwide Children's Hospital, Columbus, OH, USA.
Nat Commun ; 12(1): 4251, 2021 07 12.
Article en En | MEDLINE | ID: mdl-34253733
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Descarboxilasas de Aminoácido-L-Aromático / Mesencéfalo / Imagen por Resonancia Magnética / Terapia Genética / Técnicas de Transferencia de Gen / Dependovirus / Neuronas Dopaminérgicas / Errores Innatos del Metabolismo de los Aminoácidos Tipo de estudio: Prognostic_studies Límite: Child / Child, preschool / Female / Humans / Male Idioma: En Año: 2021 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Search on Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Descarboxilasas de Aminoácido-L-Aromático / Mesencéfalo / Imagen por Resonancia Magnética / Terapia Genética / Técnicas de Transferencia de Gen / Dependovirus / Neuronas Dopaminérgicas / Errores Innatos del Metabolismo de los Aminoácidos Tipo de estudio: Prognostic_studies Límite: Child / Child, preschool / Female / Humans / Male Idioma: En Año: 2021 Tipo del documento: Article