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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....
Autores principales: | , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2014
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Materias: | |
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 |