Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates
Open accessAbstract
Adeno-associated viral (AAV) vectors have become the primary delivery agent for somatic gene transfer into the central nervous system (CNS). To date, AAV-mediated gene delivery to the CNS is based on serotypes 1-9, with efficient gene transfer to neurons only-selective and widespread transduction of glial cells have not been observed. Recently, additional endogenous AAVs have been isolated from nonhuman primate tissues. In this study, transduction obtained with AAV serotypes bb2, cy5, rh20, rh39, and rh43 was compared to that obtained with AAV8, another nonhuman primate isolate previously shown to perform well in mammalian brain. Titer-matched vectors encoding the enhanced green fluorescent protein (EGFP) reporter, driven by the constitutive CAG promoter, were injected into the hippocampus, striatum, or substantia nigra (SN) of adult rats. More widespread neuronal transduction was observed following infusion of cy5, rh20, and rh39 than observed with AAV8. Of interest, preferential transduction of astrocytes was observed with rh43. To optimize glial transduction, vector stocks driven by cell-specific promoters were generated-widespread and targeted transduction of astrocytes and oligodendrocytes was observed using rh43 and AAV8, driven by the glial fibrillary acidic protein (GFAP) and myelin basic protein (MBP) promoters, expanding the utility of AAV for modeling and treating diseases involving glial cell pathology.
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Preclinical proof-of-concept studies in rodents have shown that using cellular promoters can target AAV-mediated transgene expression to glia.
“Preclinical proof-of-concept studies showing successful modification of the AAV system towards selective transgene expression in oligodendrocytes used “neurotropic” serotypes but employed the promoter of the mouse myelin basic protein (Mbp) gene (Chen et al., 1998; Lawlor et al., 2009; von Jonquieres et al., 2013).”
From Recombinant Human Myelin-Associated Glycoprotein Promoter Drives Selective AAV-Mediated Transgene Expression in Oligodendrocytes (2016) · cites this paper as Lawlor et al., 2009
To date, the only cellular promoter used for AAV-mediated transgene expression in oligodendrocytes is the murine 1.9 kb Mbp cis-regulating element and a truncated version spanning the proximal 1.3 kb upstream region including parts of Mbp exon 1.
“To date, the only cellular promoter used for AAV-mediated transgene expression in oligodendrocytes is the murine 1.9 kb Mbp cis-regulating element and a truncated version spanning the proximal 1.3 kb upstream region including parts of Mbp exon 1 (Chen et al., 1998; Lawlor et al., 2009; von Jonquieres et al., 2013).”
From Recombinant Human Myelin-Associated Glycoprotein Promoter Drives Selective AAV-Mediated Transgene Expression in Oligodendrocytes (2016) · cites this paper as Lawlor et al., 2009
The Mbp promoters have been used independently to achieve reporter gene expression in the adult rodent CNS using AAV2, AAV8, rh39, rh20 and cy5.
“The Mbp promoters have been used independently to achieve reporter gene expression in the adult rodent CNS using AAV2, AAV8, rh39, rh20 and cy5 (Chen et al., 1998; Lawlor et al., 2009).”
From Recombinant Human Myelin-Associated Glycoprotein Promoter Drives Selective AAV-Mediated Transgene Expression in Oligodendrocytes (2016) · cites this paper as Lawlor et al., 2009
A recent pioneering study has confirmed very good vector spread following cy5-CAG-GFP delivery to the adult rodent brain, but the effects of cellular promoters were not examined.
“While a recent pioneering study has confirmed very good vector spread following cy5-CAG-GFP delivery to the adult rodent brain the effects of cellular promoters were not examined (Lawlor et al., 2009).”
From Recombinant Human Myelin-Associated Glycoprotein Promoter Drives Selective AAV-Mediated Transgene Expression in Oligodendrocytes (2016) · cites this paper as Lawlor et al., 2009
The overall transduction of oligodendrocytes by AAV is still low.
“However, the overall transduction of oligodendrocytes is still low.15–16”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [16]
Some AAV capsids exhibit a weak tropism for oligodendrocytes when a cell-specific promoter drives expression (myelin basic protein).
“Some AAV capsids exhibit a weak tropism for oligodendrocytes when a cell-specific promoter drives expression (myelin basic protein).”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [16]
Past studies have described the ability of AAV2 or AAV8 to transduce oligodendrocytes at a low efficiency, but these studies required the use of oligodendrocyte-specific promoters to prevent expression in neurons.
“Past studies have described the ability of AAV2 or AAV8 to transduce oligodendrocytes at a low efficiency, but these studies required the use of oligodendrocyte-specific promoters to prevent expression in neurons, the preferred cell type for these vectors.15,16”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [16]
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 [16]
Previous studies by other groups targeting oligodendroglia using wild-type rAAVs with expression driven by MBP promoters had low overall transduction of oligodendrocytes.
“Previous studies by other groups target oligodendroglia using wild-type rAAVs with expression driven by MBP promoters, however the overall transduction of oligodendrocytes was still low [8, 34].”
From Novel oligodendroglial alpha synuclein viral vector models of multiple system atrophy: studies in rodents and nonhuman primates (2017) · cites this paper as [34]
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- Authors
- Lawlor, P. A., Bland, R. J., Mouravlev, A., Young, D., & During, M. J.
- Affiliations
- University of Auckland (NZ)
- AgResearch (NZ)
- Corresponding author
- Lawlor, Patricia A
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2009-10
- Volume
- 17
- Issue
- 10
- Pages
- 1692-1702
- ISSN
- 1525-0016 (ISSN-L)
- PMC
- PMC2835020
- OpenAlex
- W2047388966
- Cited by
- 161
- References
- 44
- Citations by year
- 2026: 3 · 2025: 5 · 2024: 4 · 2023: 3 · 2022: 6 · 2021: 13 · 2020: 11 · 2019: 9 · 2018: 13 · 2017: 11 · 2016: 14 · 2015: 10 · 2014: 12 · 2013: 15 · 2012: 12
- FWCI
- 6.20
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Molecular Therapy (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- ResearchSpace (University of Auckland) (submittedVersion)
- Funders
- University of Pennsylvania
- National Institutes of Health
- National Institute of Neurological Disorders and Stroke (R01NS044576, NS44576)
- 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, AAV serotypes, astrocytes, oligodendrocytes, neuronal transduction, AAV8, glial fibrillary acidic protein promoter, myelin basic protein promoter, cell-specific promoters, gene delivery to the brain
- MeSH terms
- Animals, Brain, Cell Line, Electrophoresis, Polyacrylamide Gel, Genetic Vectors, Humans, Immunohistochemistry, Male, Neuroglia, Primates, Transduction, Genetic, Rats, Sprague-Dawley, Gene Transfer Techniques, Green Fluorescent Proteins, Rats
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