ArticleThe Journal of neuroscience : the official journal of the Society for Neuroscience2002
Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic L-amino acid decarboxylase, tyrosine hydroxylase, and GTP cyclohydrolase I induces sustained transgene expression, dopamine production, and functional improvement in a rat model of Parkinson's disease.
Article in The Journal of neuroscience : the official journal of the Society for Neuroscience, 2002. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 85 papers.
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Who cites it
85 citing papers in PubMed, 219 citations in OpenAlex.
- Long-Term Follow-Up of a Phase I/II Study of ProSavin, a Lentiviral Vector Gene Therapy for Parkinson's Disease.Human gene therapy. Clinical development · 2018Trial
- Platform-dependent performance of P2A and IRES linkers in multicistronic gene expression.Genes & genomics · 2026Article
- Advancement in Gene Therapy for the Treatment of Parkinson's Disease: A Comprehensive Review.Mini reviews in medicinal chemistry · 2026Review
- IRES-Mediated Translation: Expanding the Toolkits of RNA Therapy.International journal of molecular sciences · 2025Review
- Deep brain stimulation is well tolerated and effective following ProSavin® gene therapy for Parkinson's disease.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2025Article
- Recent developments in gene therapy for Parkinson's disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Genetic therapies for movement disorders - current status.Journal of neurology · 2025Review
- Neurosurgical gene therapy for central nervous system diseases.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024Review
- Advances of Genome Editing with CRISPR/Cas9 in Neurodegeneration: The Right Path towards Therapy.Biomedicines · 2023Review
- The Role of Striatal Cav1.3 Calcium Channels in Therapeutics for Parkinson's Disease.Handbook of experimental pharmacology · 2023Review
- Genetically engineered mesenchymal stem cells with dopamine synthesis for Parkinson's disease in animal models.NPJ Parkinson's disease · 2022Article
- Internal Ribosome Entry Site (IRES)-Mediated Translation and Its Potential for Novel mRNA-Based Therapy Development.Biomedicines · 2022Review
- Gene-Based Therapeutics for Parkinson's Disease.Biomedicines · 2022Review
- Gene Therapy: The Next-Generation Therapeutics and Their Delivery Approaches for Neurological Disorders.Frontiers in genome editing · 2022Review
- Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases.Molecular neurobiology · 2022Review
- Multiple Molecular Pathways Are Influenced by Progranulin in a Neuronal Cell Model-A Parallel Omics Approach.Frontiers in neuroscience · 2021Article
- Gene and Cell-Based Therapies for Parkinson's Disease: Where Are We?Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2020Review
- Extracellular Vesicles as Nanotherapeutics for Parkinson's Disease.Biomolecules · 2020Review
- Reduction of depression-like behavior in rat model induced by ShRNA targeting norepinephrine transporter in locus coeruleus.Translational psychiatry · 2020Article
- Gene-Editing Technologies Paired With Viral Vectors for Translational Research Into Neurodegenerative Diseases.Frontiers in molecular neuroscience · 2020Article
25 more citing papers are in PubMed but not listed here.
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Authors and funding
9 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Parkinson's disease (PD) is a neurodegenerative disorder characterized by the selective loss of dopaminergic neurons in the substantia nigra. This loss leads to complete dopamine depletion in the striatum and severe motor impairment. It has been demonstrated previously that a lentiviral vector system based on equine infectious anemia virus (EIAV) gives rise to highly efficient and sustained transduction of neurons in the rat brain. Therefore, a dopamine replacement strategy using EIAV has been investigated as a treatment in the 6-hydroxydopamine (6-OHDA) animal model of PD. A self-inactivating EIAV minimal lentiviral vector that expresses tyrosine hydroxylase (TH), aromatic amino acid dopa decarboxylase (AADC), and GTP cyclohydrolase 1 (CH1) in a single transcription unit has been generated. In cultured striatal neurons transduced with this vector, TH, AADC, and CH1 proteins can all be detected. After stereotactic delivery into the dopamine-denervated striatum of the 6-OHDA-lesioned rat, sustained expression of each enzyme and effective production of catecholamines were detected, resulting in significant reduction of apomorphine-induced motor asymmetry compared with control animals (p < 0.003). Expression of each enzyme in the striatum was observed for up to 5 months after injection. These data indicate that the delivery of three catecholaminergic synthetic enzymes by a single lentiviral vector can achieve functional improvement and thus open the potential for the use of this vector for gene therapy of late-stage PD patients.
Indexed as
Identifiers
12451130PMC6758736W2130275119What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.