Frameless intraputaminal delivery of gene therapy with eladocagene exuparvovec in patients with aromatic L-amino acid decarboxylase deficiency: safe and efficient results
Frameless, neuronavigated gene therapy for AADC deficiency proved both feasible and safe, with early clinical improvements observed in motor function and symptom control. This technique offers a promising alternative to frame-based methods and expands treatment options for this rare neurometabolic disorder.
Authors
- Roni Mai (ORCID: https://orcid.org/0000-0002-1376-390X)
- Ekaterina Zakharova (ORCID: https://orcid.org/0000-0002-5020-1180)
- Svetlana Mikhaylova (ORCID: https://orcid.org/0000-0002-2115-985X)
- Sergey V. Gorelikov (ORCID: https://orcid.org/0009-0008-9471-7761)
- D.A. Reshchikov (ORCID: https://orcid.org/0000-0001-8146-5501)
- Vladimir E. Popov (ORCID: https://orcid.org/0000-0003-4835-8047)
Institutions
- Pirogov Russian National Research Medical University (RU)
- Russian Children's Clinical Hospital (RU)
- Moscow Regional Scientific Research Clinical Institute. MF Vladimirsky (RU)
- Moscow Regional Research Institute of Obstetrics and Gynecology (RU)
- Research Centre for Medical Genetics (RU)
- Regional Clinical Research (US)
Publication Details
- Journal
- Child s Nervous System
- Published
- 2025-11-10
- DOI
- https://doi.org/10.1007/s00381-025-07020-y
- Citations
- 1
- Primary Topic
- Metabolism and Genetic Disorders
- Type
- article
- Field-Weighted Citation Impact
- 1.32