Nuclear accumulation of CHMP7 initiates nuclear pore complex injury and subsequent TDP-43 dysfunction in sporadic and familial ALS

  • Alyssa N. Coyne
    Brain Science Institute, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA.
  • Victoria Baskerville
    Brain Science Institute, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA.
  • Benjamin L. Zaepfel
    Biochemistry, Cellular, and Molecular Biology Graduate Program, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA.
  • Dennis W. Dickson
    Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
  • Frank Rigo
    Ionis Pharmaceuticals, Carlsbad, CA 92010, USA.
  • Frank Bennett
    Ionis Pharmaceuticals, Carlsbad, CA 92010, USA.
  • C. Patrick Lusk
    Department of Cell Biology, Yale School of Medicine, New Haven, CT 06520, USA.
  • Jeffrey D. Rothstein
    Brain Science Institute, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA.

書誌事項

公開日
2021-07-28
DOI
  • 10.1126/scitranslmed.abe1923
公開者
American Association for the Advancement of Science (AAAS)

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説明

<jats:p>Nuclear accumulation of CHMP7 leads to multiple cellular pathologies that can be mitigated via CHMP7 ASOs in familial and sporadic ALS iPSNs.</jats:p>

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