Selection of rAAV vectors that cross the human blood-brain barrier and target the central nervous system using a transwell model
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Abstract
A limitation for recombinant adeno-associated virus (rAAV)-mediated gene transfer into the central nervous system (CNS) is the low penetration of vectors across the human blood-brain barrier (BBB). High doses of intravenously delivered vector are required to reach the CNS, which has resulted in varying adverse effects. Moreover, selective transduction of various cell types might be important depending on the disorder being treated. To enhance BBB penetration and improve CNS cell selectivity, we screened an AAV capsid-shuffled library using an in vitro transwell BBB system with separate layers of human endothelial cells, primary astrocytes and/or human induced pluripotent stem cell-derived cortical neurons. After multiple passages through the transwell, we identified chimeric AAV capsids with enhanced penetration and improved transduction of astrocytes and/or neurons compared with wild-type capsids. We identified the amino acids (aa) from regions 451–470 of AAV2 associated with the capsids selected for neurons, and a combination of aa from regions 413–496 of AAV-rh10 and 538–598 of AAV3B/LK03 associated with capsids selected for astrocytes. A small interfering RNA screen identified several genes that affect transcytosis of AAV across the BBB. Our work supports the use of a human transwell system for selecting enhanced AAV capsids targeting the CNS and may allow for unraveling the underlying molecular mechanisms of BBB penetration.
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Scavenger receptors MSR1, SCARB1, and SCARA3; insulin-like growth factor receptors IGF1R and IGF2R; antibody transporting receptors FCGRT and TMEM30A; and LEPR, LRP8, CNR1, EPHA2, PTAFR, and CCR2 improve AAV transcytosis.
“They reported, for the first time, that scavenger receptors (MSR1, SCARB1, and SCARA3), insulin-like growth factor receptors (IGF1R and IGF2R), antibody transporting receptors (FCGRT and TMEM30A), as well as LEPR, LRP8, CNR1, EPHA2, PTAFR, and CCR2 improved AAV transcytosis”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [120]
The Results section reports that knocking down every listed receptor reduced transcytosis of two or more rAAVs in the transwell model. The Discussion says these receptors *may* enhance AAV transcytosis and calls for further study of their roles; the claim presents that tentative, model-specific finding as an established improvement. (LLM, conf=0.86)
Experimental — accuracy not yet measuredINSR, AGER, AAVR, M6PR, TFRC, and LRP2 receptors inhibit AAV transcytosis.
“whereas INSR, AGER, AAVR, M6PR, TFRC, and LRP2 receptors participated in inhibition of AAV transcytosis.”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [120]
The Results section says INSR, AGER, AAVR, M6PR, TFRC, and LRP2 may participate in inhibiting rAAV transcytosis in the transwell model: knocking them down increased flowthrough for two or more rAAVs. The citing sentence presents their participation as established, whereas the paper qualifies it as a possibility. (LLM, conf=0.86)
Experimental — accuracy not yet measured
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- Authors
- Song, R., Pekrun, K., Khan, T. A., Zhang, F., Paşca, S. P., & Kay, M. A.
- Affiliations
- Stanford Medicine (US)
- Corresponding author
- Kay, Mark A.
- Venue
- Molecular Therapy - Methods & Clinical Development
- Publisher
- Elsevier BV
- Published
- 2022-12
- Volume
- 27
- Pages
- 73-88
- ISSN
- 2329-0501 (ISSN-L)
- PMC
- PMC9494039
- OpenAlex
- W4294884172
- Cited by
- 36
- References
- 37
- Citations by year
- 2026: 7 · 2025: 9 · 2024: 13 · 2023: 6
- FWCI
- 4.22
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Molecular Therapy — Methods & Clinical Development (publishedVersion, cc-by-nc-nd)
- bioRxiv (Cold Spring Harbor Laboratory) (acceptedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Funders
- National Institutes of Health (R01AI116698)
- 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, AAV capsid engineering, blood-brain barrier penetration, central nervous system gene delivery, neuronal transduction, AAV2, AAVrh10, transcytosis, iPSC-derived neurons
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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