Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons
利用磁共振引导将AAV2-AADC直接递送至中脑多巴胺能神经元，治疗芳香族L-氨基酸脱羧酶缺乏症的基因疗法
期刊:Nature Communications
影响因子:15.7
doi:10.1038/s41467-021-24524-8
Pearson, Toni S; Gupta, Nalin; San Sebastian, Waldy; Imamura-Ching, Jill; Viehoever, Amy; Grijalvo-Perez, Ana; Fay, Alex J; Seth, Neha; Lundy, Shannon M; Seo, Youngho; Pampaloni, Miguel; Hyland, Keith; Smith, Erin; de Oliveira Barbosa, Gardenia; Heathcock, Jill C; Minnema, Amy; Lonser, Russell; Elder, J Bradley; Leonard, Jeffrey; Larson, Paul; Bankiewicz, Krystof S
