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The advent of AAV9 expands applications for brain and spinal cord gene delivery
Open accessAbstract
INTRODUCTION: Straightforward studies compared adeno-associated virus (AAV) serotypes to determine the most appropriate one for robust expression in the CNS. AAV9 was efficient when directly injected into the brain, but more surprisingly, AAV9 produced global expression in the brain and spinal cord after a peripheral, systemic route of administration to neonatal mice. AREAS COVERED: Topics include AAV9 gene delivery from intraparenchymal, intravenous, intrathecal and intrauterine routes of administration, and related preclinical studies and disease models. Systemic AAV9 gene transfer yields remarkably consistent neuronal expression, though only in early development. AAV9 is versatile to study neuropathological proteins: microtubule-associated protein tau and transactive response DNA-binding protein 43 kDa (TDP-43). EXPERT OPINION: AAV9 will be more widely used based on current data, although other natural serotypes and recombineered vectors may also support or improve upon wide-scale expression. A peripheral-to-central gene delivery that can affect the entire CNS without having to inject the CNS is promising for basic functional experiments, and potentially for gene therapy. Systemic or intra-cerebrospinal fluid routes of AAV9 administration should be considered for spinal muscular atrophy, lysosomal storage diseases and amyotrophic lateral sclerosis, if more neuronal expression can be achieved in adults, or if glial expression can be exploited.
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“However, somatic transgenesis in the mouse, achieved by vector delivery at P0, is instrumental for disease modeling (Dayton et al., 2012).”
From Recombinant Human Myelin-Associated Glycoprotein Promoter Drives Selective AAV-Mediated Transgene Expression in Oligodendrocytes (2016) · cites this paper as Dayton et al., 2012
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Gene Therapy: A Promising Approach to Treating Spinal Muscular Atrophy
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“AAV9 is widely used in gene therapy applications because it is considered harmless (Dayton et al. 2012; Murrey et al. 2014; Schuster et al. 2014; Hocquemiller et al. 2016), therefore this serotype might be suitable for targeting large-sized DRG neurons with large-diameter, non-noxious afferents.”
BackgroundAn Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy
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“59 Among the 13 serotypes of AAV, AAV2, AAV6, 60 AAV8, 48,61 and AAV9 47,50,62 are commonly used for gene transduction in SMA models. rAAV2 was the first vector to be effective in neuromuscular cell transduction.”
In vivo Reprogramming of Adult Somatic Cells to Pluripotency by Overexpression of Yamanaka Factors
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Genetic modulation of the HTR2A gene reduces anxiety-related behavior in mice
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Dopaminergic REST/NRSF is protective against manganese-induced neurotoxicity in mice
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“Among several serotypes of AAVs, AAV9 is highly ef fi cient for transgene expression in neurons when directly injected into the brain (46).”
BackgroundNanomedicines to treat rare neurological disorders: The case of Krabbe disease
Moore, T. L., Pannuzzo, G., Costabile, G., Palange, A. L., Spanò, R., Ferreira, M. F. M. M., Graziano, A. C. E., Decuzzi, P., & Cardile, V. (2023) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2023.115132 · Cited by 16
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“Up to date AAV9 has become the most preferable serotype for spinal cord gene transfer (9-14).”
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Low Seroprevalence of Neutralizing Antibodies Targeting Two Clade F AAV in Humans
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Suppression of pullulanase-induced cytotoxic T cell response with a dual promoter in GSD IIIa mice
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Perspective on innovative therapies for globoid cell leukodystrophy
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“Viral vectors hardly cross the BBB, with the exception of AAV9 (Dayton et al., 2012).”
BackgroundA Molecular Reporter for Monitoring Autophagic Flux in Nervous System In Vivo
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“Another possibility would be to use a different vector that is more effective in crossing the blood brain barrier, such as AAV 9 [48].”
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Egr1 is a sex-dependent regulator of neuronal chromatin, structural plasticity, and behaviour
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Viral strategies for targeting spinal neuronal subtypes in adult wild-type rodents
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“We selected neurotropic AAV serotype 9 for its broad transduction in the central nervous system (29).”
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Viral Vector Delivery of DREADDs for CNS Therapy
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Neurologic orphan diseases: Emerging innovations and role for genetic treatments
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Transdermal spinal catheter placement in piglets: Description and validation of the technique
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“However, intrathecal adminstration improves the direct effect on the target cells using lower ector doses than the one used for parenteral injections, reduces eripheral expression and can minimise the risk of a systemic mmune response (Bevan et al., 2011; Dayton et al., 2012).”
BackgroundTian, S., Liu, Y., Hong, H., & Li, S. (2025) · Molecular Neurobiology · doi:10.1007/s12035-025-05280-y · Cited by 8
“As early as 2016, the first anti-tau VHH was developed as a probe for specific detection [189].”
Intranasal delivery of shRNA to knockdown the 5HT-2A receptor enhances memory and alleviates anxiety
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“We selected the AAV9 serotype, which has been shown to be a highly ef fi cient vector for transgene expression in neurons throughout the CNS [20, 21].”
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Syndecan-4 Mediates the Cellular Entry of Adeno-Associated Virus 9
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“It is worth noting that in rodents, SDC4 expression is typical of astroglia, the main cellular targets of AAV9 in the CNS of adult mice [36,44,45,72].”
BackgroundNature-inspired IL-1 targeted therapy to treat chronic inflammatory diseases
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Robust Long-term Transduction of Common Marmoset Neuromuscular Tissue With rAAV1 and rAAV9
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“The development of various AAV serotypes, including AAV9, has further augmented the potential for targeted gene delivery, particularly in the central nervous system (CNS) (Dayton RD et al., 2012;Yamazaki Y et al., 2014).”
Metadata
- Authors
- Dayton, R. D., Wang, D. B., & Klein, R. L.
- Affiliations
- Louisiana State University Health Sciences Center Shreveport (US)
- University of Washington (US)
- Corresponding author
- Klein, Ronald L
- ORCID
- Dayton, Robert D
- Venue
- Expert Opinion on Biological Therapy
- Publisher
- Informa Healthcare
- Published
- 2012-04-20
- Volume
- 12
- Issue
- 6
- Pages
- 757-766
- ISSN
- 1471-2598 (ISSN-L)
- 1744-7682
- PMC
- PMC3361729
- OpenAlex
- W2109349667
- Cited by
- 144
- References
- 75
- Citations by year
- 2026: 7 · 2025: 13 · 2024: 11 · 2023: 13 · 2022: 8 · 2021: 4 · 2020: 7 · 2019: 7 · 2018: 8 · 2017: 9 · 2016: 14 · 2015: 12 · 2014: 14 · 2013: 13 · 2012: 4
- FWCI
- 1.68
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Expert Opinion on Biological Therapy (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institute of Neurological Disorders and Stroke (R01NS048450, NS048450)
- 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
- Health Sciences › Medicine › Genetics (clinical) › Neurogenetic and Muscular Disorders Research
- Keywords
- AAV9, adeno-associated virus serotypes, intrathecal administration, intravenous administration, intraparenchymal injection, intrauterine administration, neonatal mice, neuronal expression, tau protein, TDP-43, spinal muscular atrophy, lysosomal storage disease, amyotrophic lateral sclerosis, glial expression, gene therapy vectors
- MeSH terms
- Dependovirus, Animals, Brain, Gene Expression Regulation, Genetic Vectors, Humans, Injections, Intravenous, Injections, Intraventricular, Injections, Spinal, Neurons, Spinal Cord, Genetic Therapy, Gene Transfer Techniques
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