Recombinant Adeno-Associated Viral Vectors in the Nervous System
Abstract
Recombinant adeno-associated virus 2 (rAAV2) has been extensively used as a gene delivery vector for the nervous system. It targets primarily neurons in the nervous system and results in sustained long-term expression of transgenes. New rAAV serotypes have been characterized and demonstrated to have improved transduction efficiencies in various regions of the brain and spinal cord. This review discusses some properties of rAAV that have been studied in the nervous system such as cell tropism, duration of transgene expression, and distribution of viral transduction, as well as immunity and regulation of transgene expression issues, all of which are important for optimization of the use of rAAV in the nervous system.
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AAV2 selectively transduces neurons and its use in the CNS is well characterized.
“AAV2 selectively transduces neurons and its use in the CNS is well characterized [34–36].”
From IL-1β-driven amyloid plaque clearance is associated with an expansion of transcriptionally reprogrammed microglia (2019) · cites this paper as [34]
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References 137 · Cited by 110
- Authors
- Burger, C., Nash, K., & Mandel, R. J.
- Affiliations
- Department of Molecular Genetics and Microbiology, College of Medicine, University of Florida, Gainesville, FL 32610.
- University of Florida (US)
- Venue
- Human Gene Therapy
- Publisher
- SAGE Publications
- Published
- 2005-07
- Volume
- 16
- Issue
- 7
- Pages
- 781-791
- ISSN
- 1043-0342 (ISSN-L)
- 1557-7422
- OpenAlex
- W2060034256
- Cited by
- 110
- References
- 137
- Citations by year
- 2025: 1 · 2022: 1 · 2020: 1 · 2019: 3 · 2018: 2 · 2017: 4 · 2016: 2 · 2015: 7 · 2014: 9 · 2013: 5 · 2012: 9
- FWCI
- 5.75
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Human Gene Therapy (publishedVersion)
- PubMed (publishedVersion)
- 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
- recombinant adeno-associated virus, AAV2, gene delivery vectors, nervous system, serotypes, neuronal targeting, transgene expression duration, virus distribution, immune response, transgene expression regulation, brain, spinal cord
- MeSH terms
- Dependovirus, Animals, Central Nervous System, Gene Expression Regulation, Genetic Vectors, Mannitol, Nervous System Diseases, Transduction, Genetic, Genetic Therapy, Transgenes
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