Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons.
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Nat Commun. 2021 Jul 12;12(1):4251. doi: 10.1038/s41467-021-24524-8.
Nat Commun. 2021.
PMID: 34253733
Free PMC article.
Clinical Trial.