Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons

Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons

Aromatic L-amino acid decarboxylase deficiency (AADC) is a rare neurodevelopmental disorder. Here the authors describe a clinical trial of MR-guided delivery of AAV2-AADC for the treatment of AADC.

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Autores principales: Toni S. Pearson, Nalin Gupta, Waldy San Sebastian, Jill Imamura-Ching, Amy Viehoever, Ana Grijalvo-Perez, Alex J. Fay, Neha Seth, Shannon M. Lundy, Youngho Seo, Miguel Pampaloni, Keith Hyland, Erin Smith, Gardenia de Oliveira Barbosa, Jill C. Heathcock, Amy Minnema, Russell Lonser, J. Bradley Elder, Jeffrey Leonard, Paul Larson, Krystof S. Bankiewicz
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2021
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Science
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Acceso en línea:https://doaj.org/article/77c5fbe7e4b84dc5bdd76784b47254d0
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