Gene transfer and expression in oligodendrocytes under the control of myelin basic protein transcriptional control region mediated by adeno-associated virus
Abstract
In this study, a rAAV vector carrying a reporter gene, 'humanized' green fluorescent protein (GFP), linked to the transcriptional control region from the myelin basic protein (MBP) gene (a myelin-forming cell-specific gene) was constructed. Transduction of oligodendrocytes was carried out both in vitro and in vivo. The GFP expression was detected for at least 3 weeks in both transduced oligodendrocyte cell line (MOCH-1 cells) and primary cultures of rat oligodendrocytes. Preferential GFP expression in oligodendrocytes was observed in the primary cultures. In contrast, transduction with rAAV carrying the CMV promoter produced stronger GFP fluorescence in various cell types, with the majority of GFP-expressing cells being the astrocytes. Infusion of approximately 6 x 10(9) particles (2 x 10(5) infectious units) of rAAV-MBP-GFP into mouse brains resulted in the GFP expression specifically in white matter. The GFP protein was detected 15 days later by immunostaining, specifically in the oligodendrocytes. No astrocytes were transduced. Our studies suggested that cell types other than neurons in the central nervous system can also be transduced by rAAV using a cell-type-specific transcriptional control region or promoter. The MBP transcriptional control region might be suitable for gene therapy and other neurobiology studies requiring direct targeting to the myelinating cells.
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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 Chen et al., 1998
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 Chen et al., 1998
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 Chen et al., 1998
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 [15]
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 [15]
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 [15]
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 [8]
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- Authors
- Chen, H., McCarty, D., Bruce, A., Suzuki, K., & Suzuki, K.
- Affiliations
- University of North Carolina at Chapel Hill (US)
- ORCID
- Chen, H
- Venue
- Gene Therapy
- Published
- 1998-01-01
- Volume
- 5
- Issue
- 1
- Pages
- 50-58
- ISSN
- 0969-7128 (ISSN-L)
- 1476-5462
- OpenAlex
- W2335277478
- Cited by
- 87
- References
- 0
- Citations by year
- 2025: 2 · 2024: 3 · 2023: 1 · 2022: 3 · 2021: 5 · 2020: 2 · 2019: 1 · 2018: 2 · 2017: 2 · 2016: 3 · 2014: 3 · 2013: 3 · 2012: 3
- FWCI
- 3.79
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Gene Therapy (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Institutes of Health (5t32hd007201-18, 5r01ns024289-16)
- National Institute of Neurological Disorders and Stroke (NS 24453, R01NS024453, NS-24289)
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (T32-HD07201)
- 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, myelin basic protein, transcriptional control region, oligodendrocytes, GFP reporter, astrocytes, white matter, gene therapy vectors, viral transduction
- MeSH terms
- Dependovirus, Animals, Brain, Cell Line, Cells, Cultured, Myelin Basic Protein, Female, Genetic Vectors, Immunohistochemistry, Luminescent Proteins, Male, Mice, Inbred C57BL, Microscopy, Fluorescence, Oligodendroglia, Genetic Therapy, Gene Expression, Gene Transfer Techniques, Green Fluorescent Proteins, Mice
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