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....

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Mol Ther Methods Clin Dev
Prif Awduron: 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
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Nature Publishing Group 2014
Pynciau:
Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC4274790/
https://ncbi.nlm.nih.gov/pubmed/25541617
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2014.49
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