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
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/77c5fbe7e4b84dc5bdd76784b47254d0
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spelling	oai:doaj.org-article:77c5fbe7e4b84dc5bdd76784b47254d02021-12-02T15:33:16ZGene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons10.1038/s41467-021-24524-82041-1723https://doaj.org/article/77c5fbe7e4b84dc5bdd76784b47254d02021-07-01T00:00:00Zhttps://doi.org/10.1038/s41467-021-24524-8https://doaj.org/toc/2041-1723Aromatic 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.Toni S. PearsonNalin GuptaWaldy San SebastianJill Imamura-ChingAmy ViehoeverAna Grijalvo-PerezAlex J. FayNeha SethShannon M. LundyYoungho SeoMiguel PampaloniKeith HylandErin SmithGardenia de Oliveira BarbosaJill C. HeathcockAmy MinnemaRussell LonserJ. Bradley ElderJeffrey LeonardPaul LarsonKrystof S. BankiewiczNature PortfolioarticleScienceQENNature Communications, Vol 12, Iss 1, Pp 1-12 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q 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 Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons
description	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.
format	article
author	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
author_facet	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
author_sort	Toni S. Pearson
title	Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons
title_short	Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons
title_full	Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons
title_fullStr	Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons
title_full_unstemmed	Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons
title_sort	gene therapy for aromatic l-amino acid decarboxylase deficiency by mr-guided direct delivery of aav2-aadc to midbrain dopaminergic neurons
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/77c5fbe7e4b84dc5bdd76784b47254d0
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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

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