Long-term post-mortem studies following neurturin gene therapy in patients with advanced Parkinson’s disease
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We performed post-mortem studies on two patients with advanced Parkinson’s disease 8 and10 years following AAV2-neurturin (CERE120) gene therapy, the longest post-mortem trophic factor gene therapy cases reported to date. CERE120 was delivered to the putamen bilaterally in one case (10 years post-surgery), and to the putamen plus the substantia nigra bilaterally in the second (8 years post-surgery). In both patients there was persistent, albeit limited, neurturin expression in the putamen covering ∼3–12% of the putamen. In the putamen, dense staining of tyrosine hydroxylase-positive fibres was observed in areas that contained detectable neurturin expression. In the substantia nigra, neurturin expression was detected in 9.8–18.95% and 22.02–39% of remaining melanin-containing neurons in the patient with putamenal and combined putamenal and nigral gene delivery, respectively. Melanized neurons displayed intense tyrosine hydroxylase and RET proto-oncogene expression in nigral neurons in the patient where CERE120 was directly delivered to the nigra. There was no difference in the degree of Lewy pathology in comparison to untreated control patients with Parkinson’s disease, and α-synuclein aggregates were detected in neurons that also stained for neurturin, RET, and tyrosine hydroxylase. These changes were not associated with antiparkinsonian benefits likely due to the limited neurturin expression. This study provides the longest term evidence of persistent transgene expression following gene delivery to the CNS and the first human results when targeting both the terminal fields in the putamen as well as the originating nigral neurons.
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AAV-2 vector encoding human NRTN gene (CERE120) has been developed to potentially provide clinical benefits in Parkinson's disease.
“NRTN displays neuroprotection through enhancing dopaminergic neuronal survival and behavioural function in animal models [73, 74], and adeno-associated virus serotype 2 (AAV-2) vector encoding the human NRTN gene (CERE120) has been developed to potentially provide clinical benefits in Parkinson’s disease [75].”
From CI-994 is a dual modulator of class I HDACs and Wnt/β-catenin signaling for the treatment of Alzheimer's disease (2026) · cites this paper as [75]
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- Authors
- Chu, Y., Bartus, R. T., Manfredsson, F. P., Olanow, C. W., & Kordower, J. H.
- Affiliations
- Rush University Medical Center (US)
- RTBioconsultants, Inc, San Diego, CA 92130, USA
- Barrow Neurological Institute (US)
- Icahn School of Medicine at Mount Sinai (US)
- Corresponding author
- Kordower, Jeffrey H
- Venue
- Brain
- Publisher
- Oxford University Press (OUP)
- Published
- 2020-03-01
- Volume
- 143
- Issue
- 3
- Pages
- 960-975
- ISSN
- 0006-8950 (ISSN-L)
- 1460-2156
- PMC
- PMC7089653
- OpenAlex
- W3012801913
- Cited by
- 88
- References
- 50
- Citations by year
- 2026: 8 · 2025: 8 · 2024: 16 · 2023: 12 · 2022: 11 · 2021: 18 · 2020: 15
- FWCI
- 6.06
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Brain (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- Funders
- Michael J. Fox Foundation
- The Parkinson’s disease Foundation
- Rush Neurological Sciences
- Topics
- Health Sciences › Medicine › Neurology › Parkinson's Disease Mechanisms and Treatments
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Neurology › Neurological disorders and treatments
- Keywords
- Parkinson's disease, neurturin, post-mortem analysis, persistent transgene expression, putamen, substantia nigra, tyrosine hydroxylase, RET proto-oncogene, Lewy body pathology, alpha-synuclein aggregates, long-term outcomes
- MeSH terms
- Proto-Oncogene Mas, Aged, Aged, 80 and over, Humans, Melanins, Middle Aged, Neurons, Parkinson Disease, Putamen, Substantia Nigra, Tyrosine 3-Monooxygenase, Genetic Therapy, Case-Control Studies, Lewy Bodies, Proto-Oncogene Proteins c-ret, Neurturin, alpha-Synuclein
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