ALS Compendium

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

Year Published: 2012

Observation: Inhibiting DRB1 suppresses TDP43 toxicity in mammalian cells and primary neurons. DRB1 enzymatic activity is required for TDP43 toxicity. Lariat intron accumulation suppresses TDP43 toxicity in the cytoplasm

Comment: Suggest that decreasing Dbr1 activity could be a potential therapeutic approach for ALS

Citation: Armakola, M., Higgins, M. J., Figley, M. D., Barmada, S. J., Scarborough, E. a, Diaz, Z., … Gitler, A. D. (2012). Inhibition of RNA lariat debranching enzyme suppresses TDP-43 toxicity in ALS disease models. Nature Genetics, 44(12), 1302–9. doi:10.1038/ng.2434

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