ArticleHuman gene therapy methods2012
Restricted transgene expression in the brain with cell-type specific neuronal promoters.
Article in Human gene therapy methods, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
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Who cites it
27 citing papers in PubMed, 43 citations in OpenAlex.
- A Cre-dependent lentiviral vector for neuron subtype-specific expression of large proteins.FEBS letters · 2026Article
- Synthetic nanoparticles for cell-type specific, spatially resolved loading and export of MiRNAs in neural cells.Journal of nanobiotechnology · 2026Article
- Properties and Application of Pseudotyped Lentiviral Vectors.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Retinoic acid-related orphan receptor alpha (RORα) as a candidate multi-pathway target in Alzheimer's disease: mechanisms and therapeutic prospects.Frontiers in neuroscience · 2026Review
- New MiniPromoter Ple389 (ADORA2A) drives selective expression in medium spiny neurons in mice and non-human primates.Scientific reports · 2024Article
- Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver.Gene therapy · 2024Article
- An enhancer-AAV approach selectively targeting dentate granule cells of the mouse hippocampus.Cell reports methods · 2024Article
- Maximizing lentiviral vector gene transfer in the CNS.Gene therapy · 2021Article
- Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters.Gene therapy · 2021Article
- Animal Models of CMT2A: State-of-art and Therapeutic Implications.Molecular neurobiology · 2020Review
- Creating New β-Globin-Expressing Lentiviral Vectors by High-Resolution Mapping of Locus Control Region Enhancer Sequences.Molecular therapy. Methods & clinical development · 2020Article
- Small Alphaherpesvirus Latency-Associated Promoters Drive Efficient and Long-Term Transgene Expression in the CNS.Molecular therapy. Methods & clinical development · 2020Article
- Lentiviral mediated RPE65 gene transfer in healthy hiPSCs-derived retinal pigment epithelial cells markedly increased RPE65 mRNA, but modestly protein level.Scientific reports · 2020Article
- Enhancer-Driven Gene Expression (EDGE) Enables the Generation of Viral Vectors Specific to Neuronal Subtypes.iScience · 2020Article
- A neuroscientist's guide to transgenic mice and other genetic tools.Neuroscience and biobehavioral reviews · 2020Review
- Fluorescent Biosensors for Neurotransmission and Neuromodulation: Engineering and Applications.Frontiers in cellular neuroscience · 2019Review
- A MicroRNA-Based Gene-Targeting Tool for Virally Labeling Interneurons in the Rodent Cortex.Cell reports · 2018Article
- Optogenetic Tractography for anatomo-functional characterization of cortico-subcortical neural circuits in non-human primates.Scientific reports · 2018Article
- Article
- Strategies for targeting primate neural circuits with viral vectors.Journal of neurophysiology · 2016Review
Corrections and comments
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Authors and funding
9 authors at 1 institution in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Tissue-targeted expression is of major interest for studying the contribution of cellular subpopulations to neurodegenerative diseases. However, in vivo methods to investigate this issue are limited. Here, we report an analysis of the cell specificity of expression of fluorescent reporter genes driven by six neuronal promoters, with the ubiquitous phosphoglycerate kinase 1 (PGK) promoter used as a reference. Quantitative analysis of AcGFPnuc expression in the striatum and hippocampus of rodents showed that all lentiviral vectors (LV) exhibited a neuronal tropism; however, there was substantial diversity of transcriptional activity and cell-type specificity of expression. The promoters with the highest activity were those of the 67 kDa glutamic acid decarboxylase (GAD67), homeobox Dlx5/6, glutamate receptor 1 (GluR1), and preprotachykinin 1 (Tac1) genes. Neuron-specific enolase (NSE) and dopaminergic receptor 1 (Drd1a) promoters showed weak activity, but the integration of an amplification system into the LV overcame this limitation. In the striatum, the expression profiles of Tac1 and Drd1a were not limited to the striatonigral pathway, whereas in the hippocampus, Drd1a and Dlx5/6 showed the expected restricted pattern of expression. Regulation of the Dlx5/6 promoter was observed in a disease condition, whereas Tac1 activity was unaffected. These vectors provide safe tools that are more selective than others available, for the administration of therapeutic molecules in the central nervous system (CNS). Nevertheless, additional characterization of regulatory elements in neuronal promoters is still required.
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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.