ReviewFrontiers in molecular neuroscience2014
Systemic gene delivery to the central nervous system using Adeno-associated virus.
Review in Frontiers in molecular neuroscience, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 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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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.
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Who cites it
37 citing papers in PubMed.
- Expression and distribution of rAAV9 intrathecally administered in juvenile to adolescent mice.Gene therapy · 2025Article
- Review
- Thyroid Hormone Clearance in the Paraventricular Nucleus of Male Mice Regulates Lean Mass and Physical Activity.Neuroendocrinology · 2024Article
- Review
- Advances in AAV technology for delivering genetically encoded cargo to the nonhuman primate nervous system.Current research in neurobiology · 2023Review
- Engineered AAVs for non-invasive gene delivery to rodent and non-human primate nervous systems.Neuron · 2022Article
- Customized blood-brain barrier shuttle peptide to increase AAV9 vector crossing the BBB and augment transduction in the brain.Biomaterials · 2022Article
- Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases.Molecular neurobiology · 2022Review
- Physiological and Pathological Factors Affecting Drug Delivery to the Brain by Nanoparticles.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2021Review
- Adeno-associated virus (AAV)-based gene therapy for glioblastoma.Cancer cell international · 2021Review
- Pilot Study Assessing the Impact of Intrathecal Administration of Variants AAV-PHP.B and AAV-PHP.eB on Brain Transduction in Adult Rhesus Macaques.Frontiers in bioengineering and biotechnology · 2021Article
- Targeting Ligands Deliver Model Drug Cargo into the Central Nervous System along Autonomic Neurons.ACS nano · 2019Article
- Gene Therapy Tools for Brain Diseases.Frontiers in pharmacology · 2019Review
- Emerging Technologies for Delivery of Biotherapeutics and Gene Therapy Across the Blood-Brain Barrier.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2018Article
- In vivo methods for acute modulation of gene expression in the central nervous system.Progress in neurobiology · 2018Review
- Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents.Journal of visualized experiments : JoVE · 2018Article
- Gene Therapy Strategies to Restore ER Proteostasis in Disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Review
- Clinical management of herpes simplex virus infections: past, present, and future.F1000Research · 2018Review
- In utero delivery of rAAV2/9 induces neuronal expression of the transgene in the brain: towards new models of Parkinson's disease.Gene therapy · 2017Article
- AAV9-based gene therapy partially ameliorates the clinical phenotype of a mouse model of Leigh syndrome.Gene therapy · 2017Article
Corrections and comments
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Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Adeno-associated virus (AAV)-mediated gene delivery has emerged as an effective and safe tool for both preclinical and clinical studies of neurological disorders. The recent discovery that several serotypes are able to cross the blood-brain barrier when administered systemically has been a real breakthrough in the field of neurodegenerative diseases. Widespread transgene expression after systemic injection could spark interest as a therapeutic approach. Such strategy will avoid invasive brain surgery and allow non-focal gene therapy promising for CNS diseases affecting large portion of the brain. Here, we will review the recent results achieved through different systemic routes of injection generated in the last decade using systemic AAV-mediated delivery and propose a brief assessment of their values. In particular, we emphasize how the methods used for virus engineering could improve brain transduction after peripheral delivery.
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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.