Safety and tolerability of MRI-guided infusion of AAV2-hAADC into the mid-brain of nonhuman primate

Safety and tolerability of MRI-guided infusion of AAV2-hAADC into the mid-brain of nonhuman primate

Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, autosomal-recessive neurological disorder caused by mutations in the DDC gene that leads to an inability to synthesize catecholamines and serotonin. As a result, patients suffer compromised development, particularly in motor function....

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Bibliographic Details
Main Authors: Waldy San Sebastian, Adrian P Kells, John Bringas, Lluis Samaranch, Piotr Hadaczek, Agnieszka Ciesielska, Michael J Macayan, Phillip J Pivirotto, John Forsayeth, Sheryl Osborne, J Fraser Wright, Foad Green, Gregory Heller, Krystof S Bankiewicz
Format: Article
Language:English
Published: Elsevier 2014-01-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050116301176

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http://www.sciencedirect.com/science/article/pii/S2329050116301176

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