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Open Access Publications from the University of California

Inhibition of RNA lariat debranching enzyme suppresses TDP-43 toxicity in ALS disease models

  • Author(s): Krogan, Nevan
  • Farese, Robert
  • Finkbeiner, Steven
  • Armakola, M
  • Higgins, MJ
  • Figley, MD
  • Barmada, SJ
  • Scarborough, EA
  • Diaz, Z
  • Fang, X
  • Shorter, J
  • Krogan, NJ
  • et al.

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Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease primarily affecting motor neurons. Mutations in the gene encoding TDP-43 cause some forms of the disease, and cytoplasmic TDP-43 aggregates accumulate in degenerating neurons of

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