Axonal transport of adeno-associated viral vectors is serotype-dependent
Open accessAbstract
We have previously shown that adeno-associated virus type 2 (AAV2) undergoes anterograde axonal transport in rat and non-human primate brain. We screened other AAV serotypes for axonal transport and found that AAV6 is transported almost exclusively in a retrograde direction and, in the same way as AAV2, it is also neuron-specific in rat brain. Our findings show that axonal transport of AAV is serotype dependent and this has implications for gene therapy of neurological diseases such as Huntington's disease.
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AAV2 can undergo anterograde axonal transport in rat and primate brains.
“It has been reported that AAV2 can undergo anterograde axonal transport in rat and primate brains (Salegio et al., 2013).”
From AAV2 and AAV9 tropism and transgene expression in the mouse eye and major tissues after intravitreal and subretinal delivery (2023) · cites this paper as Salegio et al., 2013
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- Authors
- Salegio, E. A., Samaranch, L., Kells, A. P., Mittermeyer, G., San Sebastian, W., Zhou, S., Beyer, J., Forsayeth, J., & Bankiewicz, K. S.
- Affiliations
- University of California, San Francisco (US)
- Children's Hospital of Philadelphia (US)
- Corresponding author
- Bankiewicz, K S
- Venue
- Gene Therapy
- Published
- 2012-03-15
- Volume
- 20
- Issue
- 3
- Pages
- 348-352
- ISSN
- 0969-7128 (ISSN-L)
- 1476-5462
- PMC
- PMC3381869
- OpenAlex
- W2065516762
- Cited by
- 156
- References
- 19
- Citations by year
- 2026: 7 · 2025: 4 · 2024: 10 · 2023: 9 · 2022: 10 · 2021: 10 · 2020: 19 · 2019: 11 · 2018: 13 · 2017: 10 · 2016: 20 · 2015: 12 · 2014: 9 · 2013: 11 · 2012: 1
- FWCI
- 6.34
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Gene Therapy (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- eScholarship (California Digital Library) (submittedVersion)
- Funders
- National Institutes of Health
- National Institute of Neurological Disorders and Stroke (R01NS073940)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
- Keywords
- adeno-associated virus, AAV serotypes, axonal transport, retrograde transport, anterograde transport, AAV2, AAV6, neurological disorders, Huntington's disease, viral vectors
- MeSH terms
- Dependovirus, Animals, Axonal Transport, Brain, Corpus Striatum, Fluorescent Antibody Technique, Genetic Vectors, Humans, Huntington Disease, Serotyping, Species Specificity, Thalamus, Transduction, Genetic, Genetic Therapy, Rats, Sprague-Dawley, Neurodegenerative Diseases, Green Fluorescent Proteins, Rats
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