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Open Access
Peer-reviewed
Research Article
A single amino acid variant in the variable region I of AAV capsid confers liver detargeting
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Ruxiao Xing ,
Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Validation, Visualization
¶‡ Co-first authors.
Affiliation Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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Mengyao Xu ,
Roles Data curation, Formal analysis, Investigation, Methodology, Validation, Visualization, Writing – original draft
¶‡ Co-first authors.
Affiliation Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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Darcy Reil,
Roles Data curation, Formal analysis, Investigation, Methodology
Affiliation Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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April Destefano,
Roles Investigation, Methodology
Affiliation Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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Mengtian Cui,
Roles Methodology, Resources, Software
Affiliation Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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Nan Liu,
Roles Investigation, Methodology
Affiliation Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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Jialing Liang,
Roles Investigation, Methodology
Affiliation Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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Guangchao Xu,
Roles Resources
Affiliation Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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Li Luo,
Roles Resources
Affiliation Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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Meiyu Xu,
Roles Investigation, Methodology
Affiliation Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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Fang Zhang,
Roles Investigation, Methodology, Resources
Affiliation Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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Phillip W.L. Tai,
Roles Methodology, Resources, Software
Affiliation Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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Yuquan Wei,
Roles Resources
Affiliation State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu, China
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Alisha M. Gruntman,
Roles Data curation, Formal analysis, Investigation, Methodology, Resources
Affiliation Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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Terence R. Flotte,
Roles Funding acquisition, Resources
Affiliation Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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Guangping Gao ,
Roles Funding acquisition, Supervision
* E-mail: Dan.Wang@umassmed.edu (DW); Guangping.Gao@umassmed.edu (GG)
Affiliations Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America, Department of Microbiology, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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Dan Wang
Roles Conceptualization, Data curation, Formal analysis, Funding acquisition, Project administration, Supervision, Visualization, Writing – review & editing
* E-mail: Dan.Wang@umassmed.edu (DW); Guangping.Gao@umassmed.edu (GG)
Affiliations Department of Genetic and Cellular Medicine, Horae Gene Therapy Center, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America, RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, United States of America
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A single amino acid variant in the variable region I of AAV capsid confers liver detargeting
- Ruxiao Xing,
- Mengyao Xu,
- Darcy Reil,
- April Destefano,
- Mengtian Cui,
- Nan Liu,
- Jialing Liang,
- Guangchao Xu,
- Li Luo,
- Meiyu Xu
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- Published: September 17, 2025
- https://doi.org/10.1371/journal.ppat.1013533