Gene therapy improves motor and mental function of aromatic l-amino acid decarboxylase deficiency
In patients with aromatic l-amino acid decarboxylase (AADC) deficiency, a decrease in catecholamines and serotonin levels in the brain leads to developmental delay and movement disorders. The beneficial effects of gene therapy in patients from 1 to 8 years of age with homogeneous severity of disease...
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| Main Authors: | , , , , , , , , , , , , , , , |
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| Format: | Artigo |
| Language: | English |
| Published: |
2018
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| Online Access: | https://doi.org/10.1093/brain/awy331 https://academic.oup.com/brain/article-pdf/142/2/322/27664262/awy331.pdf |
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