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Antisense oligonucleotide therapy for spinocerebellar ataxia type 2

Author

Listed:
  • Daniel R. Scoles

    (University of Utah)

  • Pratap Meera

    (University of California Los Angeles)

  • Matthew D. Schneider

    (University of Utah)

  • Sharan Paul

    (University of Utah)

  • Warunee Dansithong

    (University of Utah)

  • Karla P. Figueroa

    (University of Utah)

  • Gene Hung

    (Ionis Pharmaceuticals)

  • Frank Rigo

    (Ionis Pharmaceuticals)

  • C. Frank Bennett

    (Ionis Pharmaceuticals)

  • Thomas S. Otis

    (University of California Los Angeles
    †Present address: Roche Pharma Research and Early Development, Neuroscience, Ophthalmology & Rare Diseases, Roche Innovation Center Basel, Grenzacherstrasse 124, CH-4070 Basel, Switzerland.)

  • Stefan M. Pulst

    (University of Utah)

Abstract

Antisense oligonucleotides against ATXN2 improved motor neuron function and restored firing frequency in cerebellar Purkinje cells in mouse models of spinocerebellar ataxia type 2.

Suggested Citation

  • Daniel R. Scoles & Pratap Meera & Matthew D. Schneider & Sharan Paul & Warunee Dansithong & Karla P. Figueroa & Gene Hung & Frank Rigo & C. Frank Bennett & Thomas S. Otis & Stefan M. Pulst, 2017. "Antisense oligonucleotide therapy for spinocerebellar ataxia type 2," Nature, Nature, vol. 544(7650), pages 362-366, April.
    • Handle: RePEc:nat:nature:v:544:y:2017:i:7650:d:10.1038_nature22044
    DOI: 10.1038/nature22044
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    Cited by:

    1. M. Alejandra Zeballos C. & Hayden J. Moore & Tyler J. Smith & Jackson E. Powell & Najah S. Ahsan & Sijia Zhang & Thomas Gaj, 2023. "Mitigating a TDP-43 proteinopathy by targeting ataxin-2 using RNA-targeting CRISPR effector proteins," Nature Communications, Nature, vol. 14(1), pages 1-17, December.
    2. Renata Vieira de Sá & Emma Sudria-Lopez & Marta Cañizares Luna & Oliver Harschnitz & Dianne M. A. Heuvel & Sandra Kling & Danielle Vonk & Henk-Jan Westeneng & Henk Karst & Lauri Bloemenkamp & Suzy Var, 2024. "ATAXIN-2 intermediate-length polyglutamine expansions elicit ALS-associated metabolic and immune phenotypes," Nature Communications, Nature, vol. 15(1), pages 1-25, December.
    3. Suijuan Zhong & Mengdi Wang & Luwei Huang & Youqiao Chen & Yuxin Ge & Jiyao Zhang & Yingchao Shi & Hao Dong & Xin Zhou & Bosong Wang & Tian Lu & Xiaoxi Jing & Yufeng Lu & Junjing Zhang & Xiaoqun Wang , 2023. "Single-cell epigenomics and spatiotemporal transcriptomics reveal human cerebellar development," Nature Communications, Nature, vol. 14(1), pages 1-17, December.

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