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Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons.
Pearson TS, Gupta N, San Sebastian W, Imamura-Ching J, Viehoever A, Grijalvo-Perez A, Fay AJ, Seth N, Lundy SM, Seo Y, Pampaloni M, Hyland K, Smith E, de Oliveira Barbosa G, Heathcock JC, Minnema A, Lonser R, Elder JB, Leonard J, Larson P, Bankiewicz KS. Pearson TS, et al. Among authors: de oliveira barbosa g. 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.