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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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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