ArticleMolecular therapy : the journal of the American Society of Gene Therapy2010
Differential transduction following basal ganglia administration of distinct pseudotyped AAV capsid serotypes in nonhuman primates.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 54 papers.
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Who cites it
54 citing papers in PubMed, 87 citations in OpenAlex.
- Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside.Journal of translational medicine · 2024Review
- Neurosurgical gene therapy for central nervous system diseases.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024Review
- Unraveling the role of glial cell line-derived neurotrophic factor in the treatment of Parkinson's disease.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2024Review
- Optogenetics in primate cortical networks.Frontiers in neuroanatomy · 2023Article
- Transduction characteristics of alternative adeno-associated virus serotypes in the cat brain by intracisternal delivery.Molecular therapy. Methods & clinical development · 2022Article
- Dissecting the Prefrontal Network With Pathway-Selective Manipulation in the Macaque Brain-A Review.Frontiers in neuroscience · 2022Review
- Routes of administration for adeno-associated viruses carrying gene therapies for brain diseases.Frontiers in molecular neuroscience · 2022Review
- Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery.Gene therapy · 2021Article
- Combining brain perturbation and neuroimaging in non-human primates.NeuroImage · 2021Review
- AAV1 is the optimal viral vector for optogenetic experiments in pigeons (Columba livia).Communications biology · 2021Article
- An Open Resource for Non-human Primate Optogenetics.Neuron · 2020Article
- Intra-striatal AAV2.retro administration leads to extensive retrograde transport in the rhesus macaque brain: implications for disease modeling and therapeutic development.Scientific reports · 2020Article
- Neurons under genetic control: What are the next steps towards the treatment of movement disorders?Computational and structural biotechnology journal · 2020Review
- AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Article
- Vector-mediated l-3,4-dihydroxyphenylalanine delivery reverses motor impairments in a primate model of Parkinson's disease.Brain : a journal of neurology · 2019Article
- Concise Review: Modeling Neurodegenerative Diseases with Human Pluripotent Stem Cell-Derived Microglia.Stem cells (Dayton, Ohio) · 2019Review
- Local transgene expression and whole-body transgenesis to model brain diseases in nonhuman primate.Animal models and experimental medicine · 2019Review
- A Matter of Genes: The Hurdles of Gene Therapy for Epilepsy.Epilepsy currents · 2019Article
- Adeno-Associated Virus Technologies and Methods for Targeted Neuronal Manipulation.Frontiers in neuroanatomy · 2019Review
- Advances in optogenetic and chemogenetic methods to study brain circuits in non-human primates.Journal of neural transmission (Vienna, Austria : 1996) · 2018Review
Corrections and comments
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Authors and funding
6 authors at 3 institutions in 2 countries.
Funding
Abstract
We examined the transduction efficiency of different adeno-associated virus (AAV) capsid serotypes encoding for green fluorescent protein (GFP) flanked by AAV2 inverted terminal repeats in the nonhuman primate basal ganglia as a prelude to translational studies, as well as clinical trials in patients with Parkinson's disease (PD). Six intact young adult cynomolgus monkeys received a single 10 microl injection of AAV2/1-GFP, AAV2/5-GFP, or AAV2/8-GFP pseudotyped vectors into the caudate nucleus and putamen bilaterally in a pattern that resulted in each capsid serotype being injected into at least four striatal sites. GFP immunohistochemistry revealed excellent transduction rates for each AAV pseudotype. Stereological estimates of GFP+ cells within the striatum revealed that AAV2/5-GFP transduces significantly higher number of cells than AAV2/8-GFP (P < 0.05) and there was no significant difference between AAV2/5-GFP and AAV2/1-GFP (P = 0.348). Consistent with this result, Cavalieri estimates revealed that AAV2/5-GFP resulted in a significantly larger transduction volume than AAV2/8-GFP (P < 0.05). Each pseudotype transduced striatal neurons effectively [>95% GFP+ cells colocalized neuron-specific nuclear protein (NeuN)]. The current data suggest that AAV2/5 and AAV2/1 are superior to AAV2/8 for gene delivery to the nonhuman primate striatum and therefore better candidates for therapeutic applications targeting this structure.
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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.