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Research Article

Enhancement of SMN2 Exon 7 Inclusion by Antisense Oligonucleotides Targeting the Exon

  • Yimin Hua,

    Affiliation: Cold Spring Harbor Laboratory, Cold Spring Harbor, New York, United States of America

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  • Timothy A Vickers,

    Affiliation: Isis Pharmaceuticals, Carlsbad, California, United States of America

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  • Brenda F Baker,

    Affiliation: Isis Pharmaceuticals, Carlsbad, California, United States of America

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  • C. Frank Bennett,

    Affiliation: Isis Pharmaceuticals, Carlsbad, California, United States of America

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  • Adrian R Krainer mail

    To whom correspondence should be addressed. E-mail: krainer@cshl.edu

    Affiliation: Cold Spring Harbor Laboratory, Cold Spring Harbor, New York, United States of America

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  • Published: March 13, 2007
  • DOI: 10.1371/journal.pbio.0050073

About the Authors

Yimin Hua, Adrian R Krainer
Cold Spring Harbor Laboratory, Cold Spring Harbor, New York, United States of America
Timothy A Vickers, Brenda F Baker, C. Frank Bennett
Isis Pharmaceuticals, Carlsbad, California, United States of America

Corresponding Author

Email: krainer@cshl.edu

Competing Interests

TAV, BFB, and CFB are employees of Isis Pharmaceutical Corp., the owner of the antisense oligonucleotide chemistry used in this report, and materially benefit either directly or indirectly through stock options. YH and ARK, along with their employer, Cold Spring Harbor Laboratory, could materially benefit if a therapeutic for SMA results from this work. ARK serves on the scientific advisory board of two non-profit SMA foundations.

Author Contributions

YH, TAV, BFB, CFB, and ARK conceived and designed the experiments. YH performed the experiments. YH and ARK analyzed the data. YH, TAV, BFB, CFB, and ARK contributed reagents/materials/analysis tools. YH and ARK wrote the paper.