ArticleProceedings of the National Academy of Sciences of the United States of America2009
Efficient gene therapy-based method for the delivery of therapeutics to primate cortex.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 78 papers.
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Who cites it
78 citing papers in PubMed.
- Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons.Nature communications · 2021Trial
- Intra-CNS AAV9-Molecular therapy. Advances · 2026Article
- Advancing AADC deficiency therapy through MR-guided multisite delivery of AAV2-hAADC to dopaminergic and serotonergic pathways in the brainstem.Molecular therapy. Advances · 2026Article
- Genetic therapies for neurological diseases.Pharmacological reviews · 2026Review
- A Preliminary Study on the Accuracy of MRI-Guided Thalamic Infusion of AAV2-GFP and Biodistribution Analysis Using Cryo-Fluorescence Tomography in Nonhuman Primates.Pharmaceutics · 2025Article
- Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders.International journal of molecular sciences · 2024Review
- Intravenous functional gene transfer throughout the brain of non-human primates using AAV.Research square · 2023Article
- Pre-clinical delivery of gene therapy products to the cerebrospinal fluid: challenges and considerations for clinical translation.Frontiers in molecular neuroscience · 2023Review
- Improving the Efficacy and Accessibility of Intracranial Viral Vector Delivery in Non-Human Primates.Pharmaceutics · 2022Article
- AAV gene therapy for Tay-Sachs disease.Nature medicine · 2022Article
- Convection-Enhanced Delivery in Children: Techniques and Applications.Advances and technical standards in neurosurgery · 2022Review
- Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases.Molecular neurobiology · 2022Review
- Combining brain perturbation and neuroimaging in non-human primates.NeuroImage · 2021Review
- An Open Resource for Non-human Primate Optogenetics.Neuron · 2020Article
- Using rAAV2-retro in rhesus macaques: Promise and caveats for circuit manipulation.Journal of neuroscience methods · 2020Review
- Nanomaterial based drug delivery systems for the treatment of neurodegenerative diseases.Biomaterials science · 2020Article
- Gene therapy for neurodegenerative disorders: advances, insights and prospects.Acta pharmaceutica Sinica. B · 2020Review
- Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates.Molecular therapy. Methods & clinical development · 2019Article
- Positron emission tomography reporter gene strategy for use in the central nervous system.Proceedings of the National Academy of Sciences of the United States of America · 2019Article
- Convection Enhanced Delivery of Optogenetic Adeno-associated Viral Vector to the Cortex of Rhesus Macaque Under Guidance of Online MRI Images.Journal of visualized experiments : JoVE · 2019Article
18 more citing papers are in PubMed but not listed here.
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Authors and funding
7 authors.
Funding
Abstract
Transduction of the primate cortex with adeno-associated virus (AAV)-based gene therapy vectors has been challenging, because of the large size of the cortex. We report that a single infusion of AAV2 vector into thalamus results in widespread expression of transgene in the cortex through transduction of widely dispersed thalamocortical projections. This finding has important implications for the treatment of certain genetic and neurodegenerative diseases.
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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.