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

Qin Huang, Ken Y. Chan, Isabelle G. Tobey, Yujia Alina Chan, Tim Poterba, Christine L. Boutros, Alejandro B. Balazs, Richard Daneman, Jonathan M. Bloom, Cotton Seed, View ORCID ProfileBenjamin E. Deverman
doi: https://doi.org/10.1101/538421
Qin Huang
1Stanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA
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Ken Y. Chan
1Stanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA
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Isabelle G. Tobey
1Stanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA
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Yujia Alina Chan
1Stanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA
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Tim Poterba
1Stanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA
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Christine L. Boutros
2Ragon Institute of MGH, MIT, and Harvard, Cambridge, MA
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Alejandro B. Balazs
2Ragon Institute of MGH, MIT, and Harvard, Cambridge, MA
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Richard Daneman
3Departments of Neurosciences and Pharmacology, University of California, San Diego, La Jolla, CA
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Jonathan M. Bloom
1Stanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA
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Cotton Seed
1Stanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA
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Benjamin E. Deverman
1Stanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA
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  • ORCID record for Benjamin E. Deverman
  • For correspondence: bdeverma@broadinstitute.org
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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 useful for targeting neurological diseases in humans, we sought to elucidate the host factors responsible for the CNS tropism of AAV-PHP.B vectors. Leveraging CNS tropism differences across mouse strains, we conducted a genome-wide association study, and rapidly identified and verified LY6A as an essential receptor for the AAV-PHP.B vectors in brain endothelial cells. Importantly, this newly discovered mode of AAV binding and transduction is independent of other known AAV receptors and can be imported into different cell types to confer enhanced transduction by the AAV-PHP.B vectors.

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The copyright holder for this preprint is the author/funder, who has granted bioRxiv a license to display the preprint in perpetuity. It is made available under a CC-BY-NC-ND 4.0 International license.
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Posted February 01, 2019.
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Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids
Qin Huang, Ken Y. Chan, Isabelle G. Tobey, Yujia Alina Chan, Tim Poterba, Christine L. Boutros, Alejandro B. Balazs, Richard Daneman, Jonathan M. Bloom, Cotton Seed, Benjamin E. Deverman
bioRxiv 538421; doi: https://doi.org/10.1101/538421
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Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids
Qin Huang, Ken Y. Chan, Isabelle G. Tobey, Yujia Alina Chan, Tim Poterba, Christine L. Boutros, Alejandro B. Balazs, Richard Daneman, Jonathan M. Bloom, Cotton Seed, Benjamin E. Deverman
bioRxiv 538421; doi: https://doi.org/10.1101/538421

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