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Research Article

A Lack of Premature Termination Codon Read-Through Efficacy of PTC124 (Ataluren) in a Diverse Array of Reporter Assays

  • Stuart P. McElroy mail,

    s.mcelroy@dundee.ac.uk

    Affiliation: Drug Discovery Unit, Division of Biological Chemistry and Drug Discovery, College of Life Sciences, James Black Centre, Dow Street, University of Dundee, Dundee, United Kingdom

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  • Toshifumi Nomura,

    Affiliation: Department of Dermatology, Hokkaido University Graduate School of Medicine, Sapporo, Japan

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  • Leah S. Torrie,

    Affiliation: Drug Discovery Unit, Division of Biological Chemistry and Drug Discovery, College of Life Sciences, James Black Centre, Dow Street, University of Dundee, Dundee, United Kingdom

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  • Emma Warbrick,

    Affiliation: Division of Molecular Medicine, College of Life Sciences, Medical Sciences Institute, Dow Street, University of Dundee, Dundee, United Kingdom

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  • Ulrike Gartner,

    Affiliation: Division of Molecular Medicine, College of Life Sciences, Medical Sciences Institute, Dow Street, University of Dundee, Dundee, United Kingdom

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  • Gavin Wood,

    Affiliation: Drug Discovery Unit, Division of Biological Chemistry and Drug Discovery, College of Life Sciences, James Black Centre, Dow Street, University of Dundee, Dundee, United Kingdom

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  • W. H. Irwin McLean

    Affiliation: Division of Molecular Medicine, College of Life Sciences, Medical Sciences Institute, Dow Street, University of Dundee, Dundee, United Kingdom

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  • Published: June 25, 2013
  • DOI: 10.1371/journal.pbio.1001593

About the Authors

Stuart P. McElroy, Leah S. Torrie, Gavin Wood
Drug Discovery Unit, Division of Biological Chemistry and Drug Discovery, College of Life Sciences, James Black Centre, Dow Street, University of Dundee, Dundee, United Kingdom
Toshifumi Nomura
Department of Dermatology, Hokkaido University Graduate School of Medicine, Sapporo, Japan
Emma Warbrick, Ulrike Gartner, W. H. Irwin McLean
Division of Molecular Medicine, College of Life Sciences, Medical Sciences Institute, Dow Street, University of Dundee, Dundee, United Kingdom

Corresponding Author

Email: s.mcelroy@dundee.ac.uk

Competing Interests

I have read the journal's policy and have the following conflicts. We discovered the lack of efficacy of PTC124 by conducting our own drug discovery programme to discover and develop nonsense mutation read-through agents. Subsequent to the completion of this work, WHIM initiated a partnership with Glaxo SmithKline (GSK) on a closely related project. The work described herein was conducted prior to, and independently of, any interaction with GSK. No materials or support were received from GSK and no agreements are in place with GSK concerning the execution or publication of this work, although they were at one point involved in discussions about its publication. The collaboration with GSK has now ended.

Author Contributions

The author(s) have made the following declarations about their contributions: Conceived and designed the experiments: SPM WHIM TN. Performed the experiments: SPM TN LST UG EW. Analyzed the data: SPM LST UG EW. Contributed reagents/materials/analysis tools: TN GW UG EW. Wrote the paper: SPM WHIM TN LST UG EW.