The value of CSF neurotransmitter monitoring in the outcome of gene therapy in aromatic amino acid decarboxylase (AADC) defect
Current evidence suggests CSF metabolites do not overcome the predictive value of clinical observation on clinical outcomes and should be reserved for patients unresponsive to the therapy.
Authors
- Claudia Carducci (ORCID: https://orcid.org/0000-0003-0515-9208)
- Giacomina Ricciardi (ORCID: https://orcid.org/0000-0003-0686-2499)
- Mario Mastrangelo (ORCID: https://orcid.org/0000-0001-7664-5807)
- Filippo Manti (ORCID: https://orcid.org/0000-0002-2012-8410)
- Francesca Nardecchia (ORCID: https://orcid.org/0000-0002-0808-2827)
- Vincenzo Leuzzi (ORCID: https://orcid.org/0000-0002-2314-6139)
- Francesco Pisani (ORCID: https://orcid.org/0000-0002-6853-5605)
Institutions
- Sapienza University of Rome (IT)
Publication Details
- Journal
- Parkinsonism & Related Disorders
- Published
- 2025-05-23
- DOI
- https://doi.org/10.1016/j.parkreldis.2025.107886
- Citations
- 1
- Primary Topic
- Virus-based gene therapy research
- Type
- article
- Field-Weighted Citation Impact
- 1.82
Funders
- Ministero della Salute