ArticleGene therapy2015
Noninvasive, neuron-specific gene therapy can be facilitated by focused ultrasound and recombinant adeno-associated virus.
Article in Gene therapy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 81 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
81 citing papers in PubMed, 146 citations in OpenAlex.
- Noninvasive focal gene transfer of chemogenetic proteins in the primate brain.Cell reports methods · 2026Article
- Monitoring in vivo transcription with synthetic serum markers.Nature communications · 2026Article
- Safe focused ultrasound-mediated blood-brain barrier opening is driven primarily by transient reorganization of tight junctions.Communications engineering · 2026Article
- Protamine-1 encoded recombinant adeno-associated virus for enhanced brain magnetic resonance imaging.Magnetic resonance letters · 2026Article
- Extracellular vesicles-associated AAVs for the treatment of Machado-Joseph disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Site-specific noninvasive delivery of retrograde viral vectors to the brain.Bioengineering & translational medicine · 2026Article
- Characterization of microbubble cavitation in theranostic ultrasound-mediated blood-brain barrier opening for gene delivery.Journal of controlled release : official journal of the Controlled Release Society · 2025Article
- Viral vectors in neurodegenerative diseases: immune responses and therapeutic applications.Frontiers in neurology · 2025Review
- Adeno-associated virus vector delivery to the brain: Technology advancements and clinical applications.Advanced drug delivery reviews · 2024Review
- Engineering viral vectors for acoustically targeted gene delivery.Nature communications · 2024Article
- Focused Ultrasound-Mediated Disruption of the Blood-Brain Barrier for AAV9 Delivery in a Mouse Model of Huntington's Disease.Pharmaceutics · 2024Article
- Taking the knife to neurodegeneration: a review of surgical gene therapy delivery to the CNS.Acta neurochirurgica · 2024Review
- Acoustically targeted noninvasive gene therapy in large brain volumes.Gene therapy · 2024Article
- Noninvasive focal transgene delivery with viral neuronal tracers in the marmoset monkey.Cell reports methods · 2024Article
- The role of cellular and molecular neuroimmune crosstalk in gut immunity.Cellular & molecular immunology · 2023Review
- Single-cell mapping of focused ultrasound-transfected brain.Gene therapy · 2023Article
- Evaluation of Non-invasive Optogenetic Stimulation with Transcranial Functional Ultrasound Imaging.Ultrasound in medicine & biology · 2023Article
- Low intensity focused ultrasound: a new prospect for the treatment of Parkinson's disease.Annals of medicine · 2023Review
- Using focused ultrasound to modulate microglial structure and function.Frontiers in cellular neuroscience · 2023Review
- PET imaging of focused-ultrasound enhanced delivery of AAVs into the murine brain.Theranostics · 2023Article
21 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
5 authors at 1 institution in 1 country.
Funding
Abstract
Recombinant adeno-associated virus (rAAV) has shown great promise as a potential cure for neurodegenerative diseases. The existence of the blood-brain barrier (BBB), however, hinders efficient delivery of the viral vectors. Direct infusion through craniotomy is the most commonly used approach to achieve rAAV delivery, which carries increased risks of infection and other complications. Here, we report a focused ultrasound (FUS)-facilitated noninvasive rAAV delivery paradigm that is capable of producing targeted and neuron-specific transductions. Oscillating ultrasound contrast agents (microbubbles), driven by FUS waves, temporarily 'unlock' the BBB, allowing the systemically administrated rAAVs to enter the brain parenchyma, while maintaining their bioactivity and selectivity. Taking the advantage of the neuron-specific promoter synapsin, rAAV gene expression was triggered almost exclusively (95%) in neurons of the targeted caudate-putamen region. Both behavioral assessment and histological examination revealed no significant long-term adverse effects (in the brain and several other critical organs) for this combined treatment paradigm. Results from this study demonstrated the feasibility and safety for the noninvasive, targeted rAAV delivery, which might have open a new avenue in gene therapy in both preclinical and clinical settings.
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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.