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

  title={Inhibition of RNA lariat debranching enzyme suppresses TDP-43 toxicity in ALS disease models},
  author={Maria Armakola and Matthew J. Higgins and Matthew D. Figley and Sami J Barmada and Emily A. Scarborough and Zamia Diaz and Xiaodong Fang and James Shorter and Nevan J. Krogan and Steven Finkbeiner and Robert V Farese and Aaron D Gitler},
  booktitle={Nature Genetics},
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 most individuals with ALS. Thus, strategies aimed at targeting the toxicity of cytoplasmic TDP-43 aggregates may be effective. Here, we report results from two genome-wide loss-of-function TDP-43 toxicity suppressor screens in yeast. The… CONTINUE READING
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