ArticleGene therapy2014
Intranasal administration of plasmid DNA nanoparticles yields successful transfection and expression of a reporter protein in rat brain.
Article in Gene therapy, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
22 citing papers in PubMed, 48 citations in OpenAlex.
- Redefining intranasal brain delivery: from nasal entry to meaningful parenchymal and cellular exposure.Drug delivery · 2026Review
- Recent Advances in the Non-viral Delivery of Genes to Central Nervous System Disorders.Cellular and molecular neurobiology · 2026Review
- Intranasal Drug Delivery: Exploiting Nasal Anatomy for Enhanced Therapeutic Outcomes.Current pharmaceutical design · 2026Review
- Recent Advances in Nose-to-Brain Gene Delivery for Central Nervous System Disorders.Pharmaceutics · 2025Review
- Nose-to-Brain (N2B) Delivery: An Alternative Route for the Delivery of Biologics in the Management and Treatment of Central Nervous System Disorders.Pharmaceutics · 2023Review
- Key Features in the Design and Function of Nanocarriers for Intranasal Administration of Gene Therapy in Huntington Disease.Journal of nanotechnology and nanomaterials · 2023Article
- Towards novel nano-based vaccine platforms for SARS-CoV-2 and its variants of concern: Advances, challenges and limitations.Journal of drug delivery science and technology · 2022Review
- Gene Targeting to the Cerebral Cortex Following Intranasal Administration of Polyplexes.Pharmaceutics · 2022Article
- Re-examination of Peptide-Sequence-Dependent Gene Expression of Cysteine-Installed Pegylated Oligolysine/DNA Complexes.ACS omega · 2022Article
- Advances in the design and development of SARS-CoV-2 vaccines.Military Medical Research · 2021Review
- How Far Are Non-Viral Vectors to Come of Age and Reach Clinical Translation in Gene Therapy?International journal of molecular sciences · 2021Review
- Stimuli Responsive, Programmable DNA Nanodevices for Biomedical Applications.Frontiers in chemistry · 2021Review
- Management of Traumatic Brain Injury: From Present to Future.Antioxidants (Basel, Switzerland) · 2020Review
- Ophthalmic Administration of a DNA Plasmid Harboring the Murine Tph2 Gene: Evidence of Recombinant Tph2-FLAG in Brain Structures.Molecular biotechnology · 2020Article
- Focused ultrasound enhanced intranasal delivery of brain derived neurotrophic factor produces neurorestorative effects in a Parkinson's disease mouse model.Scientific reports · 2019Article
- Intranasal Delivery of pGDNF DNA Nanoparticles Provides Neuroprotection in the Rat 6-Hydroxydopamine Model of Parkinson's Disease.Molecular neurobiology · 2019Article
- Chitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brain.Journal of drug delivery science and technology · 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
- Article
- Pathophysiology Associated with Traumatic Brain Injury: Current Treatments and Potential Novel Therapeutics.Cellular and molecular neurobiology · 2017Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Viral vectors are a commonly used method for gene therapy because of their highly efficient transduction of cells. However, many vectors have a small genetic capacity, and their potential for immunogenicity can limit their usefulness. Moreover, for disorders of the central nervous system (CNS), the need for invasive surgical delivery of viruses to the brain also detracts from their clinical applicability. Here, we show that intranasal delivery of unimolecularly compacted DNA nanoparticles (DNA NPs), which consist of single molecules of plasmid DNA encoding enhanced green fluorescent protein (eGFP) compacted with 10 kDa polyethylene glycol (PEG)-substituted lysine 30-mers (CK30PEG10k), successfully transfect cells in the rat brain. Direct eGFP fluorescence microscopy, eGFP-immunohistochemistry (IHC) and eGFP-ELISA all demonstrated eGFP protein expression 2 days after intranasal delivery. eGFP-positive cells were found throughout the rostral-caudal axis of the brain, most often adjacent to capillary endothelial cells. This localization provides evidence for distribution of the nasally administered DNA NPs via perivascular flow. These results are the first report that intranasal delivery of DNA NPs can bypass the blood-brain barrier and transfect and express the encoded protein in the rat brain, affording a non-invasive approach for gene therapy of CNS disorders.
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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.