Gene therapy in the putamen for curing AADC deficiency and Parkinson's disease
This commentary provides an overview of the putamen as an established target site for gene therapy in treating aromatic l ‐amino acid decarboxylase (AADC) deficiency and Parkinson’s disease, two debilitating neurological disorders that involve motor dysfunction caused by dopamine deficiencies. The n...
Gespeichert in:
Veröffentlicht in: | EMBO molecular medicine 2021-09, Vol.13 (9), p.e14712-n/a |
---|---|
Hauptverfasser: | , , , , , , , , , |
Format: | Artikel |
Sprache: | eng |
Schlagworte: | |
Online-Zugang: | Volltext |
Tags: |
Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
|
Zusammenfassung: | This commentary provides an overview of the putamen as an established target site for gene therapy in treating aromatic
l
‐amino acid decarboxylase (AADC) deficiency and Parkinson’s disease, two debilitating neurological disorders that involve motor dysfunction caused by dopamine deficiencies. The neuroanatomy and the function of the putamen in motor control provide good rationales for targeting this brain structure. Additionally, the efficacy and safety of intraputaminal gene therapy demonstrate that restoration of dopamine synthesis in the putamen by using low doses of adeno‐associated viral vector serotype 2 to deliver the hAADC gene is well tolerated. This restoration leads to sustained improvements in motor and nonmotor symptoms of AADC deficiency and improved uptake and conversion of exogenous
l
‐DOPA into dopamine in Parkinson’s patients.
Graphical Abstract
This Commentary provides an overview of putamen‐targeted gene therapies for treating aromatic
l
‐amino acid decarboxylase (AADC) deficiency and Parkinson’s disease. |
---|---|
ISSN: | 1757-4676 1757-4684 1757-4684 |
DOI: | 10.15252/emmm.202114712 |