ReviewPharmaceutical research2007
Blood-brain barrier transport of non-viral gene and RNAi therapeutics.
Review in Pharmaceutical research, 2007. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 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
28 citing papers in PubMed, 84 citations in OpenAlex.
- Trojan Horses: A Secret Route for Nanomedicines.Current pharmaceutical biotechnology · 2025Review
- Navigating the intricate in-vivo journey of lipid nanoparticles tailored for the targeted delivery of RNA therapeutics: a quality-by-design approach.Journal of nanobiotechnology · 2024Review
- miRNA and antisense oligonucleotide-based α-synuclein targeting as disease-modifying therapeutics in Parkinson's disease.Frontiers in pharmacology · 2022Review
- Understanding In Vivo Fate of Nucleic Acid and Gene Medicines for the Rational Design of Drugs.Pharmaceutics · 2021Review
- The Promise and Challenges of Developing miRNA-Based Therapeutics for Parkinson's Disease.Cells · 2020Review
- Co-delivery of GOLPH3 siRNA and gefitinib by cationic lipid-PLGA nanoparticles improves EGFR-targeted therapy for glioma.Journal of molecular medicine (Berlin, Germany) · 2019Article
- Intravenously Administered Novel Liposomes, DCL64, Deliver Oligonucleotides to Cerebellar Purkinje Cells.Cerebellum (London, England) · 2019Article
- Inhibition of glioma growth by a GOLPH3 siRNA-loaded cationic liposomes.Journal of neuro-oncology · 2018Article
- Gene Therapy for Neuropathic Pain through siRNA-IRF5 Gene Delivery with Homing Peptides to Microglia.Molecular therapy. Nucleic acids · 2018Article
- Investigational chemotherapy and novel pharmacokinetic mechanisms for the treatment of breast cancer brain metastases.Pharmacological research · 2018Review
- The Race of 10 Synthetic RNAi-Based Drugs to the Pharmaceutical Market.Pharmaceutical research · 2017Review
- Current Experimental Studies of Gene Therapy in Parkinson's Disease.Frontiers in aging neuroscience · 2017Review
- Lipopolyplex for Therapeutic Gene Delivery and Its Application for the Treatment of Parkinson's Disease.Frontiers in aging neuroscience · 2016Review
- Bypassing the blood-brian barrier using established skull base reconstruction techniques.World journal of otorhinolaryngology - head and neck surgery · 2015Review
- Extracellular vesicles and their synthetic analogues in aging and age-associated brain diseases.Biogerontology · 2015Review
- Blood brain barrier: a challenge for effectual therapy of brain tumors.BioMed research international · 2015Review
- MicroRNAs in brain metastases: potential role as diagnostics and therapeutics.International journal of molecular sciences · 2014Review
- Methods for gene transfer to the central nervous system.Advances in genetics · 2014Review
- Immunoconjugates and long circulating systems: origins, current state of the art and future directions.Advanced drug delivery reviews · 2013Review
- Immunologic privilege in the central nervous system and the blood-brain barrier.Journal of cerebral blood flow and metabolism : official journal of the International Society of Cerebral Blood Flow and Metabolism · 2013Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The development of gene- and RNA interference (RNAi)-based therapeutics represents a challenge for the drug delivery field. The global brain distribution of DNA genes, as well as the targeting of specific regions of the brain, is even more complicated because conventional delivery systems, i.e. viruses, have poor diffusion in brain when injected in situ and do not cross the blood-brain barrier (BBB), which is only permeable to lipophilic molecules of less than 400 Da. Recent advances in the "Trojan Horse Liposome" (THL) technology applied to the transvascular non-viral gene therapy of brain disorders presents a promising solution to the DNA/RNAi delivery obstacle. The THL is comprised of immunoliposomes carrying either a gene for protein replacement or small hairpin RNA (shRNA) expression plasmids for RNAi effect, respectively. The THL is engineered with known lipids containing polyethyleneglycol (PEG), which stabilizes its structure in vivo in circulation. The tissue target specificity of THL is given by conjugation of approximately 1% of the PEG residues to peptidomimetic monoclonal antibodies (MAb) that bind to specific endogenous receptors (i.e. insulin and transferrin receptors) located on both the BBB and the brain cellular membranes, respectively. These MAbs mediate (a) receptor-mediated transcytosis of the THL complex through the BBB, (b) endocytosis into brain cells and (c) transport to the brain cell nuclear compartment. The present review presents an overview of the THL technology and its current application to gene therapy and RNAi, including experimental models of Parkinson's disease and brain tumors.
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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.