ArticleMolecular therapy : the journal of the American Society of Gene Therapy2009
Long-term transgene expression in the central nervous system using DNA nanoparticles.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 41 papers.
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.
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Who cites it
41 citing papers in PubMed, 79 citations in OpenAlex.
- Tissue-specific gene delivery approaches.Bioengineering & translational medicine · 2026Review
- Gene augmentation for autosomal dominant retinitis pigmentosa using rhodopsin genomic loci nanoparticles in the P23HGene therapy · 2023Article
- Review
- Prospects of Non-Coding Elements in Genomic DNA Based Gene Therapy.Current gene therapy · 2022Review
- Cell-Based Delivery Approaches for DNA-Binding Domains to the Central Nervous System.Current neuropharmacology · 2021Review
- Rhodopsin Genomic Loci DNA Nanoparticles Improve Expression and Rescue of Retinal Degeneration in a Model for Retinitis Pigmentosa.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Article
- Effects of Preweaning Manganese in Combination with Adult Striatal Dopamine Lesions on Monoamines, BDNF, TrkB, and Cognitive Function in Sprague-Dawley Rats.Neurotoxicity research · 2019Article
- Intranasal Delivery of pGDNF DNA Nanoparticles Provides Neuroprotection in the Rat 6-Hydroxydopamine Model of Parkinson's Disease.Molecular neurobiology · 2019Article
- Novel Focused Ultrasound Gene Therapy Approach Noninvasively Restores Dopaminergic Neuron Function in a Rat Parkinson's Disease Model.Nano letters · 2017Article
- Destination Brain: the Past, Present, and Future of Therapeutic Gene Delivery.Journal of neuroimmune pharmacology : the official journal of the Society on NeuroImmune Pharmacology · 2017Review
- Current Neurogenic and Neuroprotective Strategies to Prevent and Treat Neurodegenerative and Neuropsychiatric Disorders.Neuromolecular medicine · 2015Review
- Highly PEGylated DNA Nanoparticles Provide Uniform and Widespread Gene Transfer in the Brain.Advanced healthcare materials · 2015Article
- Hydroxyl PAMAM dendrimer-based gene vectors for transgene delivery to human retinal pigment epithelial cells.Nanoscale · 2015Article
- Age and lesion-induced increases of GDNF transgene expression in brain following intracerebral injections of DNA nanoparticles.Neuroscience · 2015Article
- Highly compacted pH-responsive DNA nanoparticles mediate transgene silencing in experimental glioma.Journal of materials chemistry. B · 2014Article
- Intranasal administration of plasmid DNA nanoparticles yields successful transfection and expression of a reporter protein in rat brain.Gene therapy · 2014Article
- DNA nanoparticle-mediated thymulin gene therapy prevents airway remodeling in experimental allergic asthma.Journal of controlled release : official journal of the Controlled Release Society · 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
- Long distance directional growth of dopaminergic axons along pathways of netrin-1 and GDNF.Experimental neurology · 2013Article
- GDNF gene delivery via a 2-(dimethylamino)ethyl methacrylate based cyclized knot polymer for neuronal cell applications.ACS chemical neuroscience · 2013Article
Corrections and comments
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Authors and funding
9 authors at 4 institutions in 1 country.
Funding
Abstract
This study demonstrates proof of concept for delivery and expression of compacted plasmid DNA in the central nervous system. Plasmid DNA was compacted with polyethylene glycol substituted lysine 30-mer peptides, forming rod-like nanoparticles with diameters between 8 and 11 nm. Here we show that an intracerebral injection of compacted DNA can transfect both neurons and glia, and can produce transgene expression in the striatum for up to 8 weeks, which was at least 100-fold greater than intracerebral injections of naked DNA plasmids. Bioluminescent imaging (BLI) of injected animals at the 11th postinjection week revealed significantly higher transgene activity in animals receiving compacted DNA plasmids when compared to animals receiving naked DNA. There was minimal evidence of brain inflammation. Intrastriatal injections of a compacted plasmid encoding for glial cell line-derived neurotrophic factor (pGDNF) resulted in a significant overexpression of GDNF protein in the striatum 1-3 weeks after injection.
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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.