Focal Delivery of AAV2/1-transgenes Into the Rat Brain by Localized Ultrasound-induced BBB Opening
Open accessAbstract
Delivery of drugs and macromolecules to the central nervous system (CNS) is hindered by the blood-brain barrier (BBB). Several approaches have been used to overcome this hindrance to facilitate the treatment of various CNS diseases. We now present results showing that chimeric adeno-associated virus 2/1 (AAV2/1) particles containing the coding region for the LacZ gene are efficiently delivered into the rat brain upon intravenous (IV) administration after BBB opening by focused ultrasound in the presence of vascular acoustic resonators. We show that the transgene is correctly and efficiently expressed in cells located in the neighborhood of the insonated focus, especially in the vicinity of small vessels and capillaries. Histochemical LacZ staining allows the identification of large amounts of cells expressing the enzymatically active protein. Using double immunofluorescence (IF) with antibodies against tubulinIII and bacterial LacZ, we identified these cells to be mostly neurons. A small proportion of the transduced cells was recognized as glial cells, reacting positive in the IF with antibodies against astrocytic markers. These results demonstrate that our approach allows a very specific, localized, and efficient expression of intravenously administered transgenes in the brain of rats upon ultrasound-induced BBB opening.Molecular Therapy - Nucleic Acids (2013) 2, e73; doi:10.1038/mtna.2012.64; published online 19 February 2013.
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AAV1, AAV2, or chimeric AAV1/2 vectors, which showed low BBB-crossing per se, successfully penetrated into directed brain regions after FUS-MB treatment.
“It was reported that IV infusion of AAV1, AAV2 or chimeric AAV1/2 vector, which showed low BBB-crossing per se, successfully penetrated into directed brain regions after FUS-MB treatment [140–142].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [142]
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- Authors
- Alonso, A., Reinz, E., Leuchs, B., Kleinschmidt, J., Fatar, M., Geers, B., Lentacker, I., Hennerici, M. G., de Smedt, S. C., & Meairs, S.
- Affiliations
- Heidelberg University (DE)
- German Cancer Research Center (DE)
- Ghent University (BE)
- Corresponding author
- Alonso, Angelika
- Venue
- Molecular Therapy - Nucleic Acids
- Publisher
- Elsevier BV
- Published
- 2013
- Volume
- 2
- Pages
- e73
- ISSN
- 2162-2531 (ISSN-L)
- PMC
- PMC3586801
- OpenAlex
- W2062620218
- Cited by
- 93
- References
- 40
- Citations by year
- 2026: 4 · 2025: 4 · 2024: 5 · 2023: 4 · 2022: 5 · 2021: 14 · 2020: 11 · 2019: 4 · 2018: 9 · 2017: 7 · 2016: 4 · 2015: 14 · 2014: 6 · 2013: 2
- FWCI
- 4.79
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Molecular Therapy — Nucleic Acids (publishedVersion, cc-by-nc-nd)
- Europe PMC (PubMed Central) (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- Ghent University Academic Bibliography (Ghent University) (submittedVersion, cc-by-nc-nd)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Funders
- European Commission
- Topics
- Physical Sciences › Engineering › Biomedical Engineering › Ultrasound and Hyperthermia Applications
- Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Keywords
- focused ultrasound, blood-brain barrier opening, gene delivery to the brain, beta-galactosidase expression, neurons, astrocytes, CNS gene therapy
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