ArticleScientific reports2020
Intra-striatal AAV2.retro administration leads to extensive retrograde transport in the rhesus macaque brain: implications for disease modeling and therapeutic development.
Article in Scientific reports, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 51 papers, 2 of them syntheses that pooled it.
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Who cites it
51 citing papers in PubMed, 2 syntheses or guidelines pooled it, 76 citations in OpenAlex.
- AAV Gene Therapy Drug Development and Translation of Engineered Ocular and Neurotropic Capsids: A Systematic Review Using Natural Language Processing.Clinical and translational science · 2025Pooled it
- Advances and Challenges in Gene Therapy for Neurodegenerative Diseases: A Systematic Review.International journal of molecular sciences · 2024Pooled it
- Retrograde transduction of dopaminergic cells in substantia nigra of the rhesus monkey.Scientific reports · 2026Article
- Clinical GDNF delivery methods for Parkinson's disease.Journal of Parkinson's disease · 2026Review
- A Novel Long-range Excitatory Neural Circuit from the Magnocellular Red Nucleus to Spinal Dorsal Horn Facilitates Neuropathic Pain-like Behaviors in Male Mice.Neuroscience bulletin · 2026Article
- Synthetic serum markers enable noninvasive monitoring of gene expression in primate brains.Neuron · 2026Article
- Genetically Modified Primate Models for Brain Disorder Research.Neuroscience bulletin · 2026Review
- Retrograde transduction of dopaminergic cells in substantia nigra of rhesus monkey.bioRxiv : the preprint server for biology · 2026Article
- Retrograde optogenetics reveals sensorimotor convergence within a corticotectal pathway of non-human primates.Current biology : CB · 2026Article
- Selecting an appropriate stress model of depression in rodents.World journal of psychiatry · 2025Review
- Functional vs anatomical cortico-striatal connectivity in the macaque brain.Translational psychiatry · 2025Article
- Longitudinal investigation of structural and resting-state effective connectivity alterations in a non-human primate model of Huntington's disease.Neurobiology of disease · 2025Article
- Retrograde Optogenetics Reveals Functional Convergence within a Corticotectal Pathway of Non-Human Primates.bioRxiv : the preprint server for biology · 2025Article
- Transcriptomic diversity of amygdalar subdivisions across humans and nonhuman primates.Science advances · 2025Article
- Targeted spiral ganglion neuron degeneration in parvalbumin-Cre neonatal mice.Molecular therapy. Methods & clinical development · 2025Article
- Fluorescence detection of dopamine signaling to the primate striatum in relation to stimulus-reward associations.Proceedings of the National Academy of Sciences of the United States of America · 2025Article
- Transcriptomic landscape of mammalian ventral pallidum at single-cell resolution.Science advances · 2024Article
- Stage-dependent role of interhemispheric pathway for motor recovery in primates.Nature communications · 2024Article
- Recombinant Adeno-Associated Virus Vectors for Gene Therapy of the Central Nervous System: Delivery Routes and Clinical Aspects.Biomedicines · 2024Review
- Mesoscopic calcium imaging in a head-unrestrained male non-human primate using a lensless microscope.Nature communications · 2024Article
Corrections and comments
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Authors and funding
5 authors at 2 institutions in 1 country.
Funding
Abstract
Recently, AAV2.retro, a new capsid variant capable of efficient retrograde transport in brain, was generated in mice using a directed evolution approach. However, it remains unclear to what degree transport will be recapitulated in the substantially larger and more complex nonhuman primate (NHP) brain. Here, we compared the biodistribution of AAV2.retro with its parent serotype, AAV2, in adult macaques following delivery into the caudate and putamen, brain regions which comprise the striatum. While AAV2 transduction was primarily limited to the injected brain regions, AAV2.retro transduced cells in the striatum and in dozens of cortical and subcortical regions with known striatal afferents. We then evaluated the capability of AAV2.retro to deliver disease-related gene cargo to biologically-relevant NHP brain circuits by packaging a fragment of human mutant HTT, the causative gene mutation in Huntington's disease. Following intra-striatal delivery, pathological mHTT-positive protein aggregates were distributed widely among cognitive, motor, and limbic cortico-basal ganglia circuits. Together, these studies demonstrate strong retrograde transport of AAV2.retro in NHP brain, highlight its utility in developing novel NHP models of brain disease and suggest its potential for querying circuit function and delivering therapeutic genes in the brain, particularly where treating dysfunctional circuits, versus single brain regions, is warranted.
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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.