Cited by 235Virus-based gene therapy researchBarrier Structure and Function StudiesRNA Interference and Gene Delivery

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Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids

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Huang, Qin, Chan, Ken Y., Tobey, Isabelle G., Chan, Yujia Alina, Poterba, Tim, Boutros, Christine L., Balazs, Alejandro B., Daneman, Richard, Bloom, Jonathan M., Seed, Cotton, Deverman, Benjamin E.·2019·PLOS ONE

DOI 10.1371/journal.pone.0225206PMC PMC6855452

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Abstract

The engineered AAV-PHP.B family of adeno-associated virus efficiently delivers genes throughout the mouse central nervous system. To guide their application across disease models, and to inspire the development of translational gene therapy vectors for targeting neurological diseases in humans, we sought to elucidate the host factors responsible for the CNS tropism of the AAV-PHP.B vectors. Leveraging CNS tropism differences across 13 mouse strains, we systematically determined a set of genetic variants that segregate with the permissivity phenotype, and rapidly identified LY6A as an essential receptor for the AAV-PHP.B vectors. Interfering with LY6A by CRISPR/Cas9-mediated Ly6a disruption or with blocking antibodies reduced transduction of mouse brain endothelial cells by AAV-PHP.eB, while ectopic expression of Ly6a increased AAV-PHP.eB transduction of HEK293T and CHO cells by 30-fold or more. Importantly, we demonstrate that this newly discovered mode of AAV binding and transduction can occur independently of other known AAV receptors. These findings illuminate the previously reported species- and strain-specific tropism characteristics of the AAV-PHP.B vectors and inform ongoing efforts to develop next-generation AAV vehicles for human CNS gene therapy.

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Authors: Huang, Q., Chan, K. Y., Tobey, I. G., Chan, Y. A., Poterba, T., Boutros, C. L., Balazs, A. B., Daneman, R., Bloom, J. M., Seed, C., & Deverman, B. E.
Affiliations: Broad Institute (US)
Ragon Institute of MGH, MIT and Harvard (US)
University of California San Diego (US)
Corresponding author: Deverman, Benjamin E.
ORCID: Huang, Qin
Chan, Ken Y.
Tobey, Isabelle G.
Chan, Yujia Alina
Boutros, Christine L.
Balazs, Alejandro B.
Daneman, Richard
Bloom, Jonathan M.
Deverman, Benjamin E.
Venue: PLOS ONE
Publisher: Public Library of Science (PLoS)
Published: 2019-11-14
Volume: 14
Issue: 11
Pages: e0225206
ISSN: 1932-6203 (ISSN-L)
DOI: 10.1371/journal.pone.0225206
PMC: PMC6855452
OpenAlex: W2986362275
Cited by: 234
References: 40
Citations by year: 2026: 28 · 2025: 33 · 2024: 39 · 2023: 30 · 2022: 34 · 2021: 38 · 2020: 28 · 2019: 4
FWCI: 15.67
Type: journal article
Language: en
Open access: gold
doi.org
Licence: cc-by
creativecommons.org/licenses/by/4.0/ (vor)
Locations: PLoS ONE (publishedVersion, cc-by)
bioRxiv (Cold Spring Harbor Laboratory) (acceptedVersion, cc-by-nc-nd)
PubMed Central (publishedVersion, cc-by)
PubMed (publishedVersion)
DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
Figshare (submittedVersion, cc-by)
Funders: Stanley Center for Psychiatric Research, Broad Institute
National Institute of Neurological Disorders and Stroke (UG3NS111689)
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: blood-brain barrier, gene therapy vectors, receptor identification, mouse strain differences, brain endothelial cells, neurological disorders
MeSH terms: Dependovirus, Animals, Antigens, Ly, Blood-Brain Barrier, Brain, Cell Line, Genetic Vectors, Humans, Membrane Proteins, Neurons, Transduction, Genetic, Genetic Variation, Gene Transfer Techniques, Tropism, Transgenes, Mice

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Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids

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