Dopaminergic restoration of prefrontal cortico-putaminal network in gene therapy for aromatic l-amino acid decarboxylase deficiency.
aromatic l-amino acid decarboxylase deficiency
cortico-putaminal network
dopamine restoration
gene therapy
Journal
Brain communications
ISSN: 2632-1297
Titre abrégé: Brain Commun
Pays: England
ID NLM: 101755125
Informations de publication
Date de publication:
2021
2021
Historique:
accepted:
28
02
2021
entrez:
23
8
2021
pubmed:
24
8
2021
medline:
24
8
2021
Statut:
epublish
Résumé
Aromatic l-amino acid decarboxylase (AADC) is an essential dopamine-synthesizing enzyme. In children with AADC deficiency, the gene delivery of AADC into the putamen, which functionally interacts with cortical regions, was found to improve motor function and ameliorate dystonia. However, how the restoration of dopamine in the putamen in association with cortico-putaminal networks leads to therapeutic effects remains unclear. Here, we examined neuroimaging data of eight patients with AADC deficiency (five males and three females, age range 4-19 years) who received the AADC gene therapy of the bilateral putamen in an open-label phase 1/2 study. Using high-resolution positron emission tomography with a specific AADC tracer, 6-[
Identifiants
pubmed: 34423296
doi: 10.1093/braincomms/fcab078
pii: fcab078
pmc: PMC8374966
doi:
Types de publication
Journal Article
Langues
eng
Pagination
fcab078Informations de copyright
© The Author(s) (2021). Published by Oxford University Press on behalf of the Guarantors of Brain.
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