Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain
Open accessAbstract
Recombinant adeno-associated viral (AAV) vectors can transduce cells of the CNS, resulting in long-term expression. AAV vector transduction varies depending on the serotype used and the region of the brain injected. AAV serotypes 7, 8, 9, and Rh10 have recently become available, but the transduction capabilities of these serotypes within the CNS have not been determined. We show that AAV 7, 8, 9, and Rh10 vectors expressing cDNA for a lysosomal enzyme transduce neurons, but not astrocytes or oligodendrocytes, in the cortex, striatum, hippocampus, and thalamus. Although all of the vectors contained the same genome, there were markedly different transduction patterns that could be due only to the differences in capsid proteins. The AAV 9 vector was found to undergo vector genome transport to distal neuronal cell bodies via known axonal pathways. This facilitated the distribution of enzyme, resulting in correction of lysosomal storage lesions in regions of a diseased brain that would not be corrected if the genome were not transported.
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After direct CNS infusion, almost all of the known AAV serotypes exhibit a dominant neuronal tropism.
“after direct CNS infusion, almost all of the known AAV serotypes exhibit a dominant neuronal tropism”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [12]
No AAV capsid has been described in the literature to exhibit a primary oligodendrocyte tropism when a constitutive promoter drives gene expression.
“No adeno-associated virus (AAV) capsid has been described in the literature to exhibit a primary oligodendrocyte tropism when a constitutive promoter drives gene expression”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [12]
Very few differences exist in the cellular tropism among AAV serotypes when a constitutive promoter drives gene expression.
“very few differences exist in the cellular tropism, when a constitutive promoter drives gene expression.”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [12]
The vast majority of AAV vectors, such as AAV1, 2, 5, 6, 8 or 9, exhibit a dominant neuronal tropism in the central nervous system.
“The vast majority of AAV vectors, such as AAV1, 2, 5, 6, 8 or 9, exhibit a dominant neuronal tropism in the central nervous system.”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [12]
Substantial neuronal tropism appears to be a central feature across widely diverse AAV capsid structures.
“Clearly, substantial neuronal tropism appears to be a central feature across widely diverse AAV capsid structures.”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [12]
AAV8 and AAV9 exhibit a dominant neuronal tropism after direct injection into the rodent striatum as well as in other brain structures.
“AAV8 and AAV9 exhibit a dominant neuronal tropism after direct injection into the rodent striatum9,13,16,27 as well as in other brain structures.12,28,29”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [12]
Serotype vectors with robust neuronal tropism and volumetric spread, such as AAV9, AAV8, and AAV1, are used for brain parenchyma administration and could aid with neurological illnesses that predominantly affect a specific brain region (e.g., Parkinson's disease).
“Serotype vectors with robust neuronal tropism and volumetric spread, such as AAV9, AAV8, and AAV1, are used for this type of administration [80], and could certainly aid with neurological illnesses that predominantly affect a specific brain region (e.g.Parkinson's disease [81]).”
From Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders (2022) · cites this paper as [80]
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- Authors
- Cearley, C. N., & Wolfe, J. H.
- Affiliations
- Children's Hospital of Philadelphia (US)
- Corresponding author
- Wolfe, John H.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2006-03
- Volume
- 13
- Issue
- 3
- Pages
- 528-537
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W1996706043
- Cited by
- 409
- References
- 35
- Citations by year
- 2026: 14 · 2025: 14 · 2024: 21 · 2023: 20 · 2022: 18 · 2021: 24 · 2020: 24 · 2019: 24 · 2018: 25 · 2017: 23 · 2016: 26 · 2015: 27 · 2014: 30 · 2013: 20 · 2012: 19
- FWCI
- 9.02
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Molecular Therapy (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- Funders
- National Institute of Diabetes and Digestive and Kidney Diseases (DK 46637, P30DK047757, DK 47757, R01DK046637, DK63973, R01DK063973)
- National Institute of Neurological Disorders and Stroke (R01NS038690, NS-38690)
- 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 vectors, capsid protein, lysosomal storage disease, axonal transport, neuronal targeting, brain regions, gene therapy
- MeSH terms
- Dependovirus, Animals, Axons, Brain, Capsid, Disease Models, Animal, Genetic Vectors, Glucuronidase, Humans, Mice, Inbred C3H, RNA, Messenger, Serotyping, Transduction, Genetic, Genetic Therapy, Mucopolysaccharidosis VII, Virus Assembly, Capsid Proteins, Mice
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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