ArticleJournal of controlled release : official journal of the Controlled Release Society2016
Targeted gene transfer to the brain via the delivery of brain-penetrating DNA nanoparticles with focused ultrasound.
Article in Journal of controlled release : official journal of the Controlled Release Society, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 83 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
83 citing papers in PubMed, 1 synthesis or guideline pooled it, 148 citations in OpenAlex.
- MRI Guided Focused Ultrasound-Mediated Delivery of Therapeutic Cells to the Brain: A Review of the State-of-the-Art Methodology and Future Applications.Frontiers in neurology · 2021Pooled it
- Focused Ultrasound is Poised to Take Non-Viral Gene Therapies to the Blood-Brain Barrier - and Beyond.Cell biomaterials · 2026Article
- Sonoselective Transfection of Glioma Endothelium.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Advancements in Gene Delivery using Nucleic Acid Loaded Nanoparticles for Region Specific Delivery in Alzheimer's Disease.Molecular neurobiology · 2026Review
- Delivery of monoclonal antibodies to the brain: the impact of nanocarrier structure.Drug delivery and translational research · 2026Article
- Data-Driven Feedback Identifies Focused Ultrasound Exposure Regimens for Improved Nanotheranostic Targeting of the Brain.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Overcoming the Blood-Brain Barrier: Advanced Strategies in Targeted Drug Delivery for Neurodegenerative Diseases.Pharmaceutics · 2025Review
- Ultrasound-responsive nanoparticles for imaging and therapy of brain tumors.Materials today. Bio · 2025Review
- Data-driven feedback augments ultrasound nanotheranostics in brain tumors.bioRxiv : the preprint server for biology · 2025Article
- Ultrasound-Assisted HAdvanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
- Discovery and therapeutic exploitation of Master Regulatory miRNAs in Glioblastoma.bioRxiv : the preprint server for biology · 2025Article
- Gene Therapy Techniques and Delivery Methods (Review).Sovremennye tekhnologii v meditsine · 2025Review
- Development of Brain Permeable Drugs and Novel Strategies to Overcome the Brain Barriers for Treatment Purposes.Current pharmaceutical design · 2025Review
- Article
- Focused ultrasound on the substantia nigra enables safe neurotensin-polyplex nanoparticle-mediated gene delivery to dopaminergic neurons intranasally and by blood circulation.Discover nano · 2024Article
- Solid tumor treatment via augmentation of bioactive C6 ceramide levels with thermally ablative focused ultrasound.Drug delivery and translational research · 2023Article
- Construction of a Collagen-like Protein Based on Elastin-like Polypeptide Fusion and Evaluation of Its Performance in Promoting Wound Healing.Molecules (Basel, Switzerland) · 2023Article
- Intraarterial Transplantation of Mitochondria After Ischemic Stroke Reduces Cerebral Infarction.Stroke (Hoboken, N.J.) · 2023Article
- Living Cells and Cell-Derived Vesicles: A Trojan Horse Technique for Brain Delivery.Pharmaceutics · 2023Review
- Single-cell mapping of focused ultrasound-transfected brain.Gene therapy · 2023Article
23 more citing papers are in PubMed but not listed here.
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 3 institutions in 1 country.
Funding
Abstract
Gene therapy holds promise for the treatment of many pathologies of the central nervous system (CNS), including brain tumors and neurodegenerative diseases. However, the delivery of systemically administered gene carriers to the CNS is hindered by both the blood-brain barrier (BBB) and the nanoporous and electrostatically charged brain extracelluar matrix (ECM), which acts as a steric and adhesive barrier. We have previously shown that these physiological barriers may be overcome by, respectively, opening the BBB with MR image-guided focused ultrasound (FUS) and microbubbles and using highly compact "brain penetrating" nanoparticles (BPN) coated with a dense polyethylene glycol corona that prevents adhesion to ECM components. Here, we tested whether this combined approach could be utilized to deliver systemically administered DNA-bearing BPN (DNA-BPN) across the BBB and mediate localized, robust, and sustained transgene expression in the rat brain. Systemically administered DNA-BPN delivered through the BBB with FUS led to dose-dependent transgene expression only in the FUS-treated region that was evident as early as 24h post administration and lasted for at least 28days. In the FUS-treated region ~42% of all cells, including neurons and astrocytes, were transfected, while less than 6% were transfected in the contralateral non-FUS treated hemisphere. Importantly, this was achieved without any sign of toxicity or astrocyte activation. We conclude that the image-guided delivery of DNA-BPN with FUS and microbubbles constitutes a safe and non-invasive strategy for targeted gene therapy to the brain.
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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.