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Open Access
Peer-reviewed
Research Article
The novel aminoglycoside, ELX-02, permits CTNSW138X translational read-through and restores lysosomal cystine efflux in cystinosis
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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
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The novel aminoglycoside, ELX-02, permits CTNSW138X translational read-through and restores lysosomal cystine efflux in cystinosis
- Emma J. Brasell,
- Lee Lee Chu,
- Murielle M. Akpa,
- Idit Eshkar-Oren,
- Iris Alroy,
- Rachel Corsini,
- Brian M. Gilfix,
- Yojiro Yamanaka,
- Pedro Huertas,
- Paul Goodyer
![PLOS](/resource/img/logo-plos.png)
- Published: December 4, 2019
- https://doi.org/10.1371/journal.pone.0223954