ArticleNeuroscience2011
Transgene expression in the striatum following intracerebral injections of DNA nanoparticles encoding for human glial cell line-derived neurotrophic factor.
Article in Neuroscience, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
18 citing papers in PubMed, 1 synthesis or guideline pooled it, 34 citations in OpenAlex.
- Nanomedicine to Overcome Current Parkinson's Treatment Liabilities: A Systematic Review.Neurotoxicity research · 2016Pooled it
- CNS Delivery of Nucleic Acid Therapeutics: Beyond the Blood-Brain Barrier and Towards Specific Cellular Targeting.Pharmaceutical research · 2023Review
- Antiparkinsonian Agents in Investigational Polymeric Micro- and Nano-Systems.Pharmaceutics · 2022Review
- Neurotrophins as Therapeutic Agents for Parkinson's Disease; New Chances From Focused Ultrasound?Frontiers in neuroscience · 2022Review
- Mechanistic Insight from Preclinical Models of Parkinson's Disease Could Help Redirect Clinical Trial Efforts in GDNF Therapy.International journal of molecular sciences · 2021Review
- On the Right Track to Treat Movement Disorders: Promising Therapeutic Approaches for Parkinson's and Huntington's Disease.Frontiers in aging neuroscience · 2020Review
- Intranasal Delivery of pGDNF DNA Nanoparticles Provides Neuroprotection in the Rat 6-Hydroxydopamine Model of Parkinson's Disease.Molecular neurobiology · 2019Article
- Pre-α-pro-GDNF and Pre-β-pro-GDNF Isoforms Are Neuroprotective in the 6-hydroxydopamine Rat Model of Parkinson's Disease.Frontiers in neurology · 2018Article
- Biodegradable brain-penetrating DNA nanocomplexes and their use to treat malignant brain tumors.Journal of controlled release : official journal of the Controlled Release Society · 2017Article
- Novel Focused Ultrasound Gene Therapy Approach Noninvasively Restores Dopaminergic Neuron Function in a Rat Parkinson's Disease Model.Nano letters · 2017Article
- The Non-Survival Effects of Glial Cell Line-Derived Neurotrophic Factor on Neural Cells.Frontiers in molecular neuroscience · 2017Review
- Age and lesion-induced increases of GDNF transgene expression in brain following intracerebral injections of DNA nanoparticles.Neuroscience · 2015Article
- Intranasal administration of plasmid DNA nanoparticles yields successful transfection and expression of a reporter protein in rat brain.Gene therapy · 2014Article
- Transport of glial cell line-derived neurotrophic factor into liposomes across the blood-brain barrier: in vitro and in vivo studies.International journal of molecular sciences · 2014Article
- Persistence of non-viral vector mediated RPE65 expression: case for viability as a gene transfer therapy for RPE-based diseases.Journal of controlled release : official journal of the Controlled Release Society · 2013Article
- A review of therapeutic prospects of non-viral gene therapy in the retinal pigment epithelium.Biomaterials · 2013Review
- Extended duration of transgene expression from pegylated POD nanoparticles enables attenuation of photoreceptor degeneration.PloS one · 2013Article
- Article
Corrections and comments
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Authors and funding
5 authors at 2 institutions in 1 country.
Funding
Abstract
A goal of our studies is to develop a potential therapeutic for Parkinson's disease (PD) by a human glial cell line-derived neurotrophic factor (hGDNF) expression plasmid administered to the rat striatum as a compacted DNA nanoparticle (DNP) and which will generate long-term hGDNF expression at biologically active levels. In the present study, we used a DNA plasmid encoding for hGDNF and a polyubiquitin C (UbC) promoter that was previously shown to have activity in both neurons and glia, but primarily in glia. A two-fold improvement was observed at the highest plasmid dose when using hGDNF DNA incorporating sequences found in RNA splice variant 1 compared with splice variant 2; of note, the splice variant 2 sequence is used in most preclinical studies. This optimized expression cassette design includes flanking scaffold matrix attachment elements (S/MARs) as well as a CpG-depleted prokaryotic domain and, where possible, eukaryotic elements. Stable long-term GDNF activity at levels 300-400% higher than baseline was observed following a single intracerebral injection. In a previous study, DNP plasmids encoding for reporter genes had been successful in generating long-term reporter transgene activity in the striatum (>365 days) and in this study produced sustained GDNF activity at the longest assessed time point (6 months).
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What 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.