학술논문
Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons
Document Type
article
Author
Toni S. Pearson; Nalin Gupta; Waldy San Sebastian; Jill Imamura-Ching; Amy Viehoever; Ana Grijalvo-Perez; Alex J. Fay; Neha Seth; Shannon M. Lundy; Youngho Seo; Miguel Pampaloni; Keith Hyland; Erin Smith; Gardenia de Oliveira Barbosa; Jill C. Heathcock; Amy Minnema; Russell Lonser; J. Bradley Elder; Jeffrey Leonard; Paul Larson; Krystof S. Bankiewicz
Source
Nature Communications, Vol 12, Iss 1, Pp 1-12 (2021)
Subject
Language
English
ISSN
2041-1723
Abstract
Aromatic L-amino acid decarboxylase deficiency (AADC) is a rare neurodevelopmental disorder. Here the authors describe a clinical trial of MR-guided delivery of AAV2-AADC for the treatment of AADC.