Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Publications | Publication: Gene therapy in the putamen for curing aromatic L-amino acid decarboxylase (AADC) deficiency and Parkinson’s disease

Publication: Gene therapy in the putamen for curing aromatic L-amino acid decarboxylase (AADC) deficiency and Parkinson’s disease

This article, published in EMBO Molecular Medicine, describes the role of the putamen as an established target site for gene therapy in treating AADC deficiency and Parkinson’s disease 

  • The rationale for targeting the putamen for gene therapy for the treatment of AADC deficiency and Parkinson’s disease is discussed
  • AADC gene therapy and the proposed mechanism of action of intraputaminal AADC gene therapy is described
  • A broad overview of the efficacy and safety of intraputaminal AADC gene therapy evaluated in clinical trials in children with AADC deficiency is presented
  • Additional sites for AADC gene therapy in AADC deficiency and Parkinson’s disease are discussed
  • Recommendations for assessing different therapeutic strategies for treating AADC deficiency are proposed

Hwu P, Kiening K, Anselm I, et al. EMBO Mol Med. 2021;e14712 

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Eladocagene exuparvovec is currently an investigational gene therapy product in the US. In the European Union Member States, Iceland, Norway, Lichtenstein, Northern Ireland, and Great Britain, eladocagene exuparvovec is indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L-amino acid decarboxylase (AADC) deficiency with a severe phenotype. 

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