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

• Chalermchai Mitrpant ,

  * E-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

  * E-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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