Allele-Specific Suppression of Mutant Huntingtin Using Antisense Oligonucleotides: Providing a Therapeutic Option for All Huntington Disease Patients

Niels H. Skotte; Amber L. Southwell; Michael E. Østergaard; Jeffrey B. Carroll; Simon C. Warby; Crystal N. Doty; Eugenia Petoukhov; Kuljeet Vaid; Holly Kordasiewicz; Andrew T. Watt; Susan M. Freier; Gene Hung; Punit P. Seth; C. Frank Bennett; Eric E. Swayze; Michael R. Hayden

doi:10.1371/journal.pone.0107434

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Peer-reviewed

Research Article

Allele-Specific Suppression of Mutant Huntingtin Using Antisense Oligonucleotides: Providing a Therapeutic Option for All Huntington Disease Patients

Niels H. Skotte,

Affiliation Centre for Molecular Medicine and Therapeutics, Child and Family Research Institute, University of British Columbia, Vancouver, British Columbia, Canada
⨯
Amber L. Southwell,

Affiliation Centre for Molecular Medicine and Therapeutics, Child and Family Research Institute, University of British Columbia, Vancouver, British Columbia, Canada
⨯
Michael E. Østergaard,

Affiliation ISIS Pharmaceuticals, Carlsbad, California, United States of America
⨯
Jeffrey B. Carroll,

Affiliation Behavioral Neuroscience Program, Department of Psychology, Western Washington University, Bellingham, Washington, United States of America
⨯
Simon C. Warby,

Affiliation Center for Advanced Research in Sleep Medicine, Department of Psychiatry, University of Montréal, Montréal, Quebec, Canada
⨯
Crystal N. Doty,

Affiliation Centre for Molecular Medicine and Therapeutics, Child and Family Research Institute, University of British Columbia, Vancouver, British Columbia, Canada
⨯
Eugenia Petoukhov,

Affiliation Centre for Molecular Medicine and Therapeutics, Child and Family Research Institute, University of British Columbia, Vancouver, British Columbia, Canada
⨯
Kuljeet Vaid,

Affiliation Centre for Molecular Medicine and Therapeutics, Child and Family Research Institute, University of British Columbia, Vancouver, British Columbia, Canada
⨯
Holly Kordasiewicz,

Affiliation ISIS Pharmaceuticals, Carlsbad, California, United States of America
⨯
Andrew T. Watt,

Affiliation ISIS Pharmaceuticals, Carlsbad, California, United States of America
⨯
Susan M. Freier,

Affiliation ISIS Pharmaceuticals, Carlsbad, California, United States of America
⨯
Gene Hung,

Affiliation ISIS Pharmaceuticals, Carlsbad, California, United States of America
⨯
Punit P. Seth,

Affiliation ISIS Pharmaceuticals, Carlsbad, California, United States of America
⨯
C. Frank Bennett,

Affiliation ISIS Pharmaceuticals, Carlsbad, California, United States of America
⨯
Eric E. Swayze,

Affiliation ISIS Pharmaceuticals, Carlsbad, California, United States of America
⨯
[ ... ],
Michael R. Hayden

* E-mail: mrh@cmmt.ubc.ca

Affiliation Centre for Molecular Medicine and Therapeutics, Child and Family Research Institute, University of British Columbia, Vancouver, British Columbia, Canada
⨯
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Allele-Specific Suppression of Mutant Huntingtin Using Antisense Oligonucleotides: Providing a Therapeutic Option for All Huntington Disease Patients

Niels H. Skotte,
Amber L. Southwell,
Michael E. Østergaard,
Jeffrey B. Carroll,
Simon C. Warby,
Crystal N. Doty,
Eugenia Petoukhov,
Kuljeet Vaid,
Holly Kordasiewicz,
Andrew T. Watt

Published: September 10, 2014
https://doi.org/10.1371/journal.pone.0107434

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Spectrins
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Single nucleotide polymorphisms
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Huntington disease
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