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Open Access
Peer-reviewed
Research Article
Toward an all-in-one recombinant adeno-associated virus vector for functionally ablating the prion gene using CRISPR-Cas technology
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Claire Verkuyl ,
Contributed equally to this work with: Claire Verkuyl, Ari Belotserkovsky
Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Supervision, Validation, Visualization, Writing – review & editing
Affiliations Tanz Centre for Research in Neurodegenerative Diseases, University of Toronto, Toronto, Ontario, Canada, Department of Laboratory Medicine & Pathobiology, University of Toronto, Toronto, Ontario, Canada
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Ari Belotserkovsky ,
Contributed equally to this work with: Claire Verkuyl, Ari Belotserkovsky
Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Software, Supervision, Validation, Visualization, Writing – review & editing
Affiliations Tanz Centre for Research in Neurodegenerative Diseases, University of Toronto, Toronto, Ontario, Canada, Department of Laboratory Medicine & Pathobiology, University of Toronto, Toronto, Ontario, Canada
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Thomas Zerbes,
Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Validation, Visualization, Writing – review & editing
Affiliations Tanz Centre for Research in Neurodegenerative Diseases, University of Toronto, Toronto, Ontario, Canada, Department of Laboratory Medicine & Pathobiology, University of Toronto, Toronto, Ontario, Canada
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Declan Williams,
Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Writing – review & editing
Affiliations Tanz Centre for Research in Neurodegenerative Diseases, University of Toronto, Toronto, Ontario, Canada, Department of Laboratory Medicine & Pathobiology, University of Toronto, Toronto, Ontario, Canada
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Medha R. Krishnan,
Roles Conceptualization, Formal analysis, Investigation, Methodology, Writing – review & editing
Affiliations Tanz Centre for Research in Neurodegenerative Diseases, University of Toronto, Toronto, Ontario, Canada, Department of Laboratory Medicine & Pathobiology, University of Toronto, Toronto, Ontario, Canada
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Sabrina Zhu,
Roles Conceptualization, Formal analysis, Investigation, Methodology, Visualization, Writing – review & editing
Affiliations Tanz Centre for Research in Neurodegenerative Diseases, University of Toronto, Toronto, Ontario, Canada, Department of Laboratory Medicine & Pathobiology, University of Toronto, Toronto, Ontario, Canada
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Sophie Grunnesjӧ,
Roles Conceptualization, Data curation, Investigation, Methodology, Validation, Visualization, Writing – review & editing
Affiliations Tanz Centre for Research in Neurodegenerative Diseases, University of Toronto, Toronto, Ontario, Canada, Department of Laboratory Medicine & Pathobiology, University of Toronto, Toronto, Ontario, Canada
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Shehab Eid,
Roles Conceptualization, Data curation, Investigation, Methodology, Supervision, Writing – review & editing
Affiliations Tanz Centre for Research in Neurodegenerative Diseases, University of Toronto, Toronto, Ontario, Canada, Department of Laboratory Medicine & Pathobiology, University of Toronto, Toronto, Ontario, Canada
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Cunjie Zhang,
Roles Conceptualization, Formal analysis, Investigation, Methodology, Software, Supervision, Writing – review & editing
Affiliation Tanz Centre for Research in Neurodegenerative Diseases, University of Toronto, Toronto, Ontario, Canada
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Wenda Zhao,
Roles Data curation, Methodology, Software, Supervision, Writing – review & editing
Affiliations Tanz Centre for Research in Neurodegenerative Diseases, University of Toronto, Toronto, Ontario, Canada, Department of Laboratory Medicine & Pathobiology, University of Toronto, Toronto, Ontario, Canada
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Leo Xu,
Roles Investigation, Methodology, Validation, Visualization, Writing – review & editing
Affiliations Tanz Centre for Research in Neurodegenerative Diseases, University of Toronto, Toronto, Ontario, Canada, Department of Laboratory Medicine & Pathobiology, University of Toronto, Toronto, Ontario, Canada
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Eleanore Lin,
Roles Investigation, Methodology, Visualization, Writing – review & editing
Affiliations Tanz Centre for Research in Neurodegenerative Diseases, University of Toronto, Toronto, Ontario, Canada, Department of Laboratory Medicine & Pathobiology, University of Toronto, Toronto, Ontario, Canada
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Teaghan O’Shea,
Roles Data curation, Methodology, Software, Validation, Visualization, Writing – review & editing
Affiliation Tanz Centre for Research in Neurodegenerative Diseases, University of Toronto, Toronto, Ontario, Canada
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Benjamin Draper,
Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Resources, Software, Visualization, Writing – review & editing
Affiliation Megadalton Solutions, Inc., and Department of Chemistry, Indiana University, Bloomington, Indiana, United States of America
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Andreas Jungman,
Roles Conceptualization, Investigation, Methodology, Writing – review & editing
Affiliations German Centre for Cardiovascular Research (DZHK), Heidelberg, Germany, Department of Internal Medicine III Cardiology, University of Heidelberg, Heidelberg, Germany
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Patrick Most,
Roles Conceptualization, Methodology, Supervision, Writing – review & editing
Affiliations German Centre for Cardiovascular Research (DZHK), Heidelberg, Germany, Department of Internal Medicine III Cardiology, University of Heidelberg, Heidelberg, Germany
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Gerold Schmitt-Ulms
Roles Conceptualization, Data curation, Funding acquisition, Investigation, Methodology, Project administration, Resources, Software, Supervision, Visualization, Writing – original draft
* E-mail: g.schmittulms@utoronto.ca
Affiliations Tanz Centre for Research in Neurodegenerative Diseases, University of Toronto, Toronto, Ontario, Canada, Department of Laboratory Medicine & Pathobiology, University of Toronto, Toronto, Ontario, Canada
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Toward an all-in-one recombinant adeno-associated virus vector for functionally ablating the prion gene using CRISPR-Cas technology
- Claire Verkuyl,
- Ari Belotserkovsky,
- Thomas Zerbes,
- Declan Williams,
- Medha R. Krishnan,
- Sabrina Zhu,
- Sophie Grunnesjӧ,
- Shehab Eid,
- Cunjie Zhang,
- Wenda Zhao
- Published: November 7, 2025
- https://doi.org/10.1371/journal.pone.0336578