Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System
Open accessAbstract
Noninvasive systemic gene delivery to the central nervous system (CNS) has largely been impeded by the blood-brain barrier (BBB). Recent studies documented widespread CNS gene transfer after intravascular delivery of recombinant adeno-associated virus 9 (rAAV9). To investigate alternative and possibly more potent rAAV vectors for systemic gene delivery across the BBB, we systematically evaluated the CNS gene transfer properties of nine different rAAVEGFP vectors after intravascular infusion in neonatal mice. Several rAAVs efficiently transduce neurons, motor neurons, astrocytes, and Purkinje cells; among them, rAAVrh.10 is at least as efficient as rAAV9 in many of the regions examined. Importantly, intravenously delivered rAAVs did not cause abnormal microgliosis in the CNS. The rAAVs that achieve stable widespread gene transfer in the CNS are exceptionally useful platforms for the development of therapeutic approaches for neurological disorders affecting large regions of the CNS as well as convenient biological tools for neuroscience research.
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AAVrh.8 better transduces neurons, astrocytes, and oligodendrocytes and has low peripheral tropism compared to AAV9 and AAVrh.10.
“AAVrh.8 was shown to better transduce neurons, astrocytes, and oligodendrocytes, while with low peripheral tropism compared to AAV9 and AAVrh.10 [126, 127].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [127]
The Discussion (page unavailable) identifies rAAVrh.10, rAAVrh.39, rAAVrh.43, and rAAV9 as effective CNS vectors, but does not assess AAVrh.8. Searches for AAVrh.8, oligodendrocytes, and peripheral tropism found no passage supporting the claimed comparison. (LLM, conf=0.76)
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- Authors
- Zhang, H., Yang, B., Mu, X., Ahmed, S. S., Su, Q., He, R., Wang, H., Mueller, C., Sena-Esteves, M., Brown, R., Xu, Z., & Gao, G.
- Affiliations
- University of Massachusetts Chan Medical School (US)
- Corresponding author
- Zhang, Hongwei
- Gao, Guangping
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2011-08
- Volume
- 19
- Issue
- 8
- Pages
- 1440-1448
- ISSN
- 1525-0016 (ISSN-L)
- PMC
- PMC3149178
- OpenAlex
- W2045600222
- Cited by
- 291
- References
- 40
- Citations by year
- 2026: 5 · 2025: 9 · 2024: 21 · 2023: 9 · 2022: 23 · 2021: 30 · 2020: 18 · 2019: 25 · 2018: 26 · 2017: 17 · 2016: 20 · 2015: 29 · 2014: 28 · 2013: 20 · 2012: 7
- FWCI
- 11.57
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Molecular Therapy (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- The Journal of the American Medical Association (JAMA) Network (American Medical Association) (submittedVersion)
- Funders
- Ohio State University
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- recombinant adeno-associated virus vectors, AAV9, blood-brain barrier, systemic gene delivery, neonatal mice, astrocytes, motor neurons, Purkinje cells, microglial activation, EGFP reporter
- MeSH terms
- Dependovirus, Animals, Animals, Newborn, Astrocytes, Blood-Brain Barrier, Central Nervous System, Genetic Vectors, Mice, Inbred C57BL, Neurons, Genetic Therapy, Gene Transfer Techniques, Mice
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