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Research Article

Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy

  • Chalermchai Mitrpant mail,

    chalermchai.mitrpant@uwa.edu.au (CM); burghes.1@osu.edu (AHMB)

    Affiliations: Centre for Neuromuscular and Neurological Disorders, The University of Western Australia, QE II Medical Centre, Perth, Western Australia, Australia, Department of Biochemistry, Faculty of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand

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  • Paul Porensky,

    Affiliation: Department of Neurological Surgery, The Wexner Ohio State University Medical Center, Columbus, Ohio, United States of America

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  • Haiyan Zhou,

    Affiliation: Dubowitz Neuromuscular Centre, Institute of Child Health and Great Ormond Street Hospital, London, United Kingdom

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  • Loren Price,

    Affiliation: Centre for Neuromuscular and Neurological Disorders, The University of Western Australia, QE II Medical Centre, Perth, Western Australia, Australia

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  • Francesco Muntoni,

    Affiliation: Dubowitz Neuromuscular Centre, Institute of Child Health and Great Ormond Street Hospital, London, United Kingdom

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  • Sue Fletcher,

    Affiliation: Centre for Neuromuscular and Neurological Disorders, The University of Western Australia, QE II Medical Centre, Perth, Western Australia, Australia

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  • Steve D. Wilton,

    Affiliation: Centre for Neuromuscular and Neurological Disorders, The University of Western Australia, QE II Medical Centre, Perth, Western Australia, Australia

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  • Arthur H. M. Burghes mail

    chalermchai.mitrpant@uwa.edu.au (CM); burghes.1@osu.edu (AHMB)

    Affiliation: Department of Molecular and Cellular Biochemistry and Neurology, The Wexner Ohio State University Medical Center, Columbus, Ohio, United States of America

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  • Published: April 22, 2013
  • DOI: 10.1371/journal.pone.0062114

About the Authors

Chalermchai Mitrpant, Loren Price, Sue Fletcher, Steve D. Wilton
Centre for Neuromuscular and Neurological Disorders, The University of Western Australia, QE II Medical Centre, Perth, Western Australia, Australia
Chalermchai Mitrpant
Department of Biochemistry, Faculty of Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand
Paul Porensky
Department of Neurological Surgery, The Wexner Ohio State University Medical Center, Columbus, Ohio, United States of America
Haiyan Zhou, Francesco Muntoni
Dubowitz Neuromuscular Centre, Institute of Child Health and Great Ormond Street Hospital, London, United Kingdom
Arthur H. M. Burghes
Department of Molecular and Cellular Biochemistry and Neurology, The Wexner Ohio State University Medical Center, Columbus, Ohio, United States of America

Corresponding Authors

Competing Interests

The authors have declared that no competing interests exist.

Author Contributions

Conceived and designed the experiments: CM SF SDW AHMB. Performed the experiments: CM PP LP. Analyzed the data: CM PP. Contributed reagents/materials/analysis tools: SF SDW AHMB. Wrote the paper: CM PP HZ FM SF SDW AHMB.