Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia

• Shengwen Zhang ,

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

  * E-mail: szhang@logicbio.com (SZ); nelsonchauck@gmail.com (BNC)

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

  https://orcid.org/0000-0001-7318-3870

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• Amy Bastille,

  Roles Methodology, Resources, Writing – review & editing

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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• Susana Gordo,

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

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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• Nikhil Ramesh,

  Roles Methodology, Resources, Writing – review & editing

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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• Jenisha Vora,

  Roles Methodology, Resources, Writing – review & editing

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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• Elizabeth McCarthy,

  Roles Methodology, Resources, Writing – review & editing

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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• Xiaohan Zhang,

  Roles Methodology, Resources, Writing – review & editing

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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• Dylan Frank,

  Roles Methodology, Resources, Writing – review & editing

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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• Chih-Wei Ko,

  Roles Methodology, Resources, Writing – review & editing

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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• Carmen Wu,

  Roles Methodology, Resources, Writing – review & editing

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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• Noel Walsh,

  Roles Methodology, Resources, Writing – review & editing

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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• Shreya Amarwani,

  Roles Methodology, Resources, Writing – review & editing

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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• Jing Liao,

  Roles Data curation, Formal analysis, Methodology, Resources, Supervision, Validation, Writing – review & editing

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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• Qiang Xiong,

  Roles Methodology, Resources, Supervision, Writing – review & editing

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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• Lauren Drouin,

  Roles Methodology, Resources, Supervision, Writing – review & editing

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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• Matthias Hebben,

  Roles Methodology, Resources, Supervision, Validation, Writing – review & editing

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

  https://orcid.org/0000-0002-2684-1153

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• Kyle Chiang,

  Roles Conceptualization, Investigation, Methodology, Project administration, Resources, Supervision, Writing – review & editing

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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•  [ ... ],
• B. Nelson Chau

  Roles Conceptualization, Data curation, Investigation, Methodology, Project administration, Resources, Supervision, Validation, Writing – review & editing

  * E-mail: szhang@logicbio.com (SZ); nelsonchauck@gmail.com (BNC)

  Affiliation LogicBio Therapeutics Inc., Lexington, MA, United States of America

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