The DOI matches its Crossref record; 75 of the 75 references it cites were found in a registry or an index.
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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1 retracted
Foust, K. D., Wang, X., McGovern, V. L., Braun, L., Bevan, A. K., Haidet, A. M., Le, T. T., Morales, P. R., Rich, M. M., Burghes, A. H. M., et al. (2010) · Nature Biotechnology · doi:10.1038/nbt.1610 · Cited by 688
RetractedUbiquitinated TDP-43 in Frontotemporal Lobar Degeneration and Amyotrophic Lateral Sclerosis
Neumann, M., Sampathu, D. M., Kwong, L. K., Truax, A. C., Micsenyi, M. C., Chou, T. T., Bruce, J., Schuck, T., Grossman, M., Clark, C. M., et al. (2006) · Science · doi:10.1126/science.1134108 · Cited by 6,576
Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection
Zincarelli, C., Soltys, S., Rengo, G., & Rabinowitz, J. E. (2008) · Molecular Therapy · doi:10.1038/mt.2008.76 · Cited by 1,490
ALS: A Disease of Motor Neurons and Their Nonneuronal Neighbors
Boillée, S., Velde, C. V., & Cleveland, D. W. (2006) · Neuron · doi:10.1016/j.neuron.2006.09.018 · Cited by 1,476
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
Foust, K. D., Nurre, E., Montgomery, C. L., Hernandez, A., Chan, C. M., & Kaspar, B. K. (2009) · Nature Biotechnology · doi:10.1038/nbt.1515 · Cited by 1,456
Clades of Adeno-Associated Viruses Are Widely Disseminated in Human Tissues
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Cited by
Sorted by citations
AAV-Mediated Gene Therapy for Research and Therapeutic Purposes
Samulski, R. J., & Muzyczka, N. (2014) · Annual Review of Virology · doi:10.1146/annurev-virology-031413-085355 · Cited by 609
Schorling, D., Pechmann, A., & Kirschner, J. (2019) · Journal of Neuromuscular Diseases · doi:10.3233/jnd-190424 · Cited by 304
Fibroblast Growth Factor-2 Signaling in Neurogenesis and Neurodegeneration
Woodbury, M. E., & Ikezu, T. (2014) · Journal of Neuroimmune Pharmacology · doi:10.1007/s11481-013-9501-5 · Cited by 266
“Gene therapy using AAV-FGF2 is similar in these regards, however FGL can be administered systemically and crosses the BBB, whereas AAV must be injected directly into the CNS, excluding AAV9 (Dayton et al. 2012).”
Körbelin, J., Dogbevia, G., Michelfelder, S., Ridder, D. A., Hunger, A., Wenzel, J., Seismann, H., Lampe, M., Bannach, J., Pasparakis, M., et al. (2016) · EMBO Molecular Medicine · doi:10.15252/emmm.201506078 · Cited by 253
“…et al, 2000; Cearley & Wolfe, 2006; de Backer et al, 2010; Markakis et al, 2010; Shen et al, 2013), and some of them (e.g., AAV9, AAVrh.8, and AAVrh.10) seem to be able to cross the mature BBB after systemic administration in adult animals (Foust et al, 2009; Dayton et al, 2012; Yang et al, 2014).”
BackgroundThévenot, E., Jordão, J. F., O’Reilly, M. A., Markham, K. A., Weng, Y., Foust, K. D., Kaspar, B. K., Hynynen, K., & Aubert, I. (2012) · Human Gene Therapy · doi:10.1089/hum.2012.013 · Cited by 202
“Advances in the AAV field have led to the selection of appropriate serotypes, exhibiting tropism for various organs (Pulicherla et al., 2011), and to the characterization of conditions that are important for infection efficiency (Gray et al., 2011; Dayton et al., 2012; Rapti et al., 2012).”
Background
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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