AAV delivery of GBA1 suppresses α-synuclein accumulation in Parkinson’s disease models and restores functions in Gaucher’s disease models

• Takuro Okai ,

  Roles Conceptualization, Formal analysis, Investigation, Methodology, Project administration, Validation, Visualization, Writing – original draft, Writing – review & editing

  * E-mail: takuro.okai@takeda.com

  Affiliation Neuroscience Drug Discovery Unit, Research, Takeda Pharmaceutical Company Limited, Fujisawa, Japan

  https://orcid.org/0009-0008-2958-9767

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• Sho Sato,

  Roles Formal analysis, Methodology, Writing – review & editing

  Affiliation Center of Excellence for Drug Metabolism, Pharmacokinetics and Modeling, Preclinical and Translational Sciences, Research, Takeda Pharmaceutical Company Limited, Fujisawa, Japan

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• Mugdha Deshpande,

  Roles Formal analysis, Investigation, Methodology, Writing – review & editing

  Affiliation Takeda Development Center Americas, Cambridge, Massachusetts, United States of America

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• Shin-ichi Matsumoto,

  Roles Formal analysis, Investigation, Methodology, Validation, Writing – review & editing

  Affiliation Center of Excellence for Drug Metabolism, Pharmacokinetics and Modeling, Preclinical and Translational Sciences, Research, Takeda Pharmaceutical Company Limited, Fujisawa, Japan

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• Miyu Nakayama,

  Roles Formal analysis, Investigation, Methodology, Validation, Writing – review & editing

  Affiliation Center of Excellence for Drug Metabolism, Pharmacokinetics and Modeling, Preclinical and Translational Sciences, Research, Takeda Pharmaceutical Company Limited, Fujisawa, Japan

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• Syunsuke Yamamoto,

  Roles Formal analysis, Methodology, Writing – review & editing

  Affiliation Center of Excellence for Drug Metabolism, Pharmacokinetics and Modeling, Preclinical and Translational Sciences, Research, Takeda Pharmaceutical Company Limited, Fujisawa, Japan

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• Bettina Strack-Logue,

  Roles Conceptualization, Writing – original draft, Writing – review & editing

  Affiliation Neuroscience Drug Discovery Unit, Research, Takeda Development Center Americas, Inc, Cambridge, Massachusetts, United States of America

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• Takeshi Hioki,

  Roles Conceptualization, Supervision, Writing – review & editing

  Affiliation Neuroscience Drug Discovery Unit, Research, Takeda Pharmaceutical Company Limited, Fujisawa, Japan

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• Maiko Tanaka,

  Roles Conceptualization, Supervision, Writing – review & editing

  Affiliation Neuroscience Drug Discovery Unit, Research, Takeda Pharmaceutical Company Limited, Fujisawa, Japan

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• Gabriele Proetzel

  Roles Conceptualization, Project administration, Supervision, Visualization, Writing – original draft, Writing – review & editing

  Affiliation Neuroscience Drug Discovery Unit, Research, Takeda Development Center Americas, Inc, Cambridge, Massachusetts, United States of America

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