ArticleMolecular therapy : the journal of the American Society of Gene Therapy2018
Extensive Transduction and Enhanced Spread of a Modified AAV2 Capsid in the Non-human Primate CNS.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 39 papers.
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Who cites it
39 citing papers in PubMed, 60 citations in OpenAlex.
- Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressingScience translational medicine · 2020Trial
- Intra-CNS AAV9-Molecular therapy. Advances · 2026Article
- Focused ultrasound expands intra-CSF AAV delivery to deep brain regions in rats and non-human primates.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Modified AAV5 capsid for improved brain biodistribution following direct injection in preclinical models.Molecular therapy. Methods & clinical development · 2025Article
- Intrathalamic delivery of adeno-associated viral vector expressing progranulin as gene therapy for GRN-related frontotemporal dementia.Molecular therapy. Methods & clinical development · 2025Article
- Delivery of AAV vectors to the superior olivary complex enables efficient adult cochlear transduction via axonal transport.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Engineered AAV capsid transport mutants overcome transduction deficiencies in the aged CNS.Molecular therapy. Nucleic acids · 2024Article
- Review
- Adeno-Associated Viral Vectors in the Treatment of Epilepsy.International journal of molecular sciences · 2024Review
- Recombinant Adeno-Associated Virus Vectors for Gene Therapy of the Central Nervous System: Delivery Routes and Clinical Aspects.Biomedicines · 2024Review
- CRISPR/Cas9 Gene Editing: A Novel Approach Towards Alzheimer's Disease Treatment.CNS & neurological disorders drug targets · 2024Review
- Development and characterization of a non-human primate model of disseminated synucleinopathy.Frontiers in neuroanatomy · 2024Article
- Genetic Approaches for Neural Circuits Dissection in Non-human Primates.Neuroscience bulletin · 2023Review
- Developing a Gene Therapy for the Treatment of Autosomal Dominant Alzheimer's Disease.Human gene therapy · 2023Article
- GDNF gene therapy for alcohol use disorder in male non-human primates.Nature medicine · 2023Article
- AAV-mediated expression of a new conformational anti-aggregated α-synuclein antibody prolongs survival in a genetic model of α-synucleinopathies.NPJ Parkinson's disease · 2023Article
- Semi-automated workflows to quantify AAV transduction in various brain areas and predict gene editing outcome for neurological disorders.Molecular therapy. Methods & clinical development · 2023Article
- Pre-clinical delivery of gene therapy products to the cerebrospinal fluid: challenges and considerations for clinical translation.Frontiers in molecular neuroscience · 2023Review
- Long-term safety and dose escalation of intracerebroventricular CLN5 gene therapy in sheep supports clinical translation for CLN5 Batten disease.Frontiers in genetics · 2023Article
- The engineered AAV2-HBKO promotes non-invasive gene delivery to large brain regions beyond ultrasound targeted sites.Molecular therapy. Methods & clinical development · 2022Article
Corrections and comments
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Authors and funding
16 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The present study was designed to characterize transduction of non-human primate brain and spinal cord with a modified adeno-associated virus serotype 2, incapable of binding to the heparan sulfate proteoglycan receptor, referred to as AAV2-HBKO. AAV2-HBKO was infused into the thalamus, intracerebroventricularly or via a combination of both intracerebroventricular and thalamic delivery. Thalamic injection of this modified vector encoding GFP resulted in widespread CNS transduction that included neurons in deep cortical layers, deep cerebellar nuclei, several subcortical regions, and motor neuron transduction in the spinal cord indicative of robust bidirectional axonal transport. Intracerebroventricular delivery similarly resulted in widespread cortical transduction, with one striking distinction that oligodendrocytes within superficial layers of the cortex were the primary cell type transduced. Robust motor neuron transduction was also observed in all levels of the spinal cord. The combination of thalamic and intracerebroventricular delivery resulted in transduction of oligodendrocytes in superficial cortical layers and neurons in deeper cortical layers. Several subcortical regions were also transduced. Our data demonstrate that AAV2-HBKO is a powerful vector for the potential treatment of a wide number of neurological disorders, and highlight that delivery route can significantly impact cellular tropism and pattern of CNS transduction.
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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.