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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Detalles Bibliográficos
Autores principales: Sebastian, Waldy San, Kells, Adrian P, Bringas, John, Samaranch, Lluis, Hadaczek, Piotr, Ciesielska, Agnieszka, Macayan, Michael J, Pivirotto, Phillip J, Forsayeth, John, Osborne, Sheryl, Wright, J Fraser, Green, Foad, Heller, Gregory, Bankiewicz, Krystof S
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2014
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4274790/
https://www.ncbi.nlm.nih.gov/pubmed/25541617
http://dx.doi.org/10.1038/mtm.2014.49

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