The novel aminoglycoside, ELX-02, permits CTNS^W138X translational read-through and restores lysosomal cystine efflux in cystinosis

• Emma J. Brasell,

  Roles Conceptualization, Data curation, Formal analysis, Methodology, Visualization, Writing – original draft, Writing – review & editing

  Affiliation McGill University, Department of Human Genetics, Montreal, Canada

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• Lee Lee Chu,

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

  Affiliation Research Institute of the McGill University Health Centre, Montreal, Canada

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• Murielle M. Akpa,

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

  Affiliation Research Institute of the McGill University Health Centre, Montreal, Canada

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• Idit Eshkar-Oren,

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

  Affiliation McGill University, Department of Experimental Medicine, Montreal, Canada

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• Iris Alroy,

  Roles Data curation, Formal analysis, Methodology, Supervision, Validation, Visualization, Writing – original draft, Writing – review & editing

  Affiliation McGill University, Department of Experimental Medicine, Montreal, Canada

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• Rachel Corsini,

  Roles Data curation, Methodology, Validation, Visualization

  Affiliation Research Institute of the McGill University Health Centre, Montreal, Canada

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• Brian M. Gilfix,

  Roles Data curation, Formal analysis, Methodology, Resources

  Affiliation Research Institute of the McGill University Health Centre, Montreal, Canada

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• Yojiro Yamanaka,

  Roles Conceptualization, Methodology, Resources, Validation

  Affiliation McGill University, Department of Human Genetics, Montreal, Canada

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• Pedro Huertas,

  Roles Conceptualization, Project administration, Resources, Supervision, Writing – original draft

  Affiliation McGill University, Department of Experimental Medicine, Montreal, Canada

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• Paul Goodyer

  Roles Conceptualization, Data curation, Funding acquisition, Investigation, Project administration, Resources, Supervision, Visualization, Writing – original draft, Writing – review & editing

  * E-mail: paul.goodyer@mcgill.ca

  Current address: The Research Institute of the McGill university Health Centre, Montreal, Quebec, Canada

  Affiliations McGill University, Department of Human Genetics, Montreal, Canada, Research Institute of the McGill University Health Centre, Montreal, Canada, Montreal Children’s Hospital, Department of Nephrology, Montreal, Canada, Eloxx Pharmaceuticals, Inc., Waltham, United States of America

  http://orcid.org/0000-0002-3442-1407

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