ArticleMovement disorders : official journal of the Movement Disorder Society2025
Intraputaminal Delivery of Adeno-Associated Virus Serotype 2-Glial Cell Line-Derived Neurotrophic Factor in Mild or Moderate Parkinson's Disease.
Article in Movement disorders : official journal of the Movement Disorder Society, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
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Who cites it
18 citing papers in PubMed.
- Genetic architecture and molecular pathogenesis of Parkinson's disease: emerging mechanisms and therapeutic targets.Metabolic brain disease · 2026Review
- Convergent Astrocytic Failure in Parkinson's Disease: A System-Level Model of Pathogenesis and Treatment.Molecular neurobiology · 2026Review
- Intraputaminal AAV9 co-delivery of AADC and GDNF in severe AADC deficiency: first-in-human pediatric dose-escalation study.Science China. Life sciences · 2026Article
- NLX-112, a Selective 5-HT1A Receptor Agonist, Upregulates GDNF and is Neuroprotective Against MPTP-Induced Nigrostriatal Degeneration in a Mouse Model of Parkinson's Disease.ACS chemical neuroscience · 2026Article
- AAV2 capsid clearance and neuronal trafficking dynamics in the central nervous system.Gene therapy · 2026Article
- A Phase 1 Study of Convection-Enhanced Delivery of Intraputaminal AAV2-GDNF in Advanced Parkinson's Disease.Movement disorders : official journal of the Movement Disorder Society · 2026Article
- Experimental change in temperament: Conversion of the low exploratory phenotype into highly explorative by overexpression of GDNF in the rat striatum.Molecular psychiatry · 2026Article
- Dual-target gene therapy in Parkinson's disease: a multicenter phase 1 trial.Nature medicine · 2026Article
- Clinical GDNF delivery methods for Parkinson's disease.Journal of Parkinson's disease · 2026Review
- Self-Assembling Short Peptide Carriers for Gene Delivery.International journal of molecular sciences · 2026Review
- Advancing AADC deficiency therapy through MR-guided multisite delivery of AAV2-hAADC to dopaminergic and serotonergic pathways in the brainstem.Molecular therapy. Advances · 2026Article
- Redefining Parkinson's Disease management: the synergistic role of neurotrophic factors and mitochondria.Cellular and molecular life sciences : CMLS · 2026Review
- Current State of the Neurotrophin-Based Pharmaceutics in the Treatment of Neurodegenerative Diseases and Neuroinflammation.Medical sciences (Basel, Switzerland) · 2025Review
- AAV2retro Enters Axons of Passage and Extensively Transduces Corticospinal Neurons After Injection into Spinal White Matter.Brain sciences · 2025Article
- Involvement of Neuroinflammation and Oxidative Stress in L-DOPA-Induced Dyskinesia in Parkinson's Disease: Role of Renin-Angiotensin System and ROCK Pathway.Antioxidants (Basel, Switzerland) · 2025Review
- Intrathecal Therapies for Neurodegenerative Diseases: A Review of Current Approaches and the Urgent Need for Advanced Delivery Systems.Biomedicines · 2025Review
- Investigational Gene Therapies for Parkinson's Disease.CNS drugs · 2025Review
- Status and future of recombinant adeno-associated virus vector manufacturing.Biotechnology progressReview
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Authors and funding
11 authors.
Funding
Abstract
backgroundGlial cell line-derived neurotrophic factor (GDNF) is required for development and survival of dopaminergic neurons. A previous trial evaluating lower-dose adeno-associated virus serotype 2-GDNF (AAV2-GDNF) bilateral intraputaminal infusion in participants with advanced Parkinson's disease (PD) achieved 26% mean putaminal coverage and was associated with stable motor features with no unexpected adverse events (AEs) over 60 months.
objectiveWe assessed safety and preliminary clinical outcomes of optimized bilateral intraputaminal infusion of a single, higher dose of AAV2-GDNF (product code AB-1005) for PD after 18 months.
methodsThis phase 1b single-arm, open-label clinical trial enrolled participants with mild (Movement Disorder Society-revised Unified Parkinson's Disease Rating Scale [MDS-UPDRS] Part III off score ≤ 32) and moderate (MDS-UPDRS Part III off score of 33-60) PD. The primary outcome was safety. Clinical outcomes were assessed using PD-specific clinical measures.
resultsEleven participants were enrolled (n = 6 mild; n = 5 moderate). Mean (±SE) putaminal coverage of AAV2-GDNF was 63% (±2%). All participants experienced treatment-emergent AEs (63 events); most were transient and perioperative. Six serious AEs in three participants were unrelated to AAV2-GDNF. At 18 months posttreatment, the mild cohort exhibited numerically stable MDS-UPDRS, motor diary, Unified Dyskinesia Rating Scale (UDysRS) scores, and levodopa equivalent daily dose (LEDD). The moderate cohort demonstrated numerical improvements in mean (±SE) MDS-UPDRS Part III off scores (-20.4 [±4.5]), motor diary off time (-1.7 [±1.1] hours), and UDysRS scores (-2.2 [±1.9]) and reduced LEDD (-257.6 [±162.2] mg).
conclusionsBilateral intraputaminal AAV2-GDNF gene therapy was well tolerated and associated with numerical stability (mild cohort) and improvement (moderate cohort) in clinical assessments at 18 months posttreatment. © 2025 AskBio Inc and The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
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