Skip to main content
Download PDF
- Main
Inhibition of RNA lariat debranching enzyme suppresses TDP-43 toxicity in ALS disease models
- Armakola, Maria;
- Higgins, Matthew J;
- Figley, Matthew D;
- Barmada, Sami J;
- Scarborough, Emily A;
- Diaz, Zamia;
- Fang, Xiaodong;
- Shorter, James;
- Krogan, Nevan J;
- Finkbeiner, Steven;
- Farese, Robert V;
- Gitler, Aaron D
Published Web Location
https://doi.org/10.1038/ng.2434Abstract
Aaron Gitler, Robert Farese Jr. and colleagues identify the RNA lariat debranching enzyme Dbr1 as a potent suppressor of TDP-43 toxicity in yeast. They further show that Dbr1 knockdown reduces TDP-43 toxicity in mammalian cells, identifying this enzyme as a possible therapeutic target in amyotrophic lateral sclerosis and other diseases marked by TDP-43 accumulation.
Many UC-authored scholarly publications are freely available on this site because of the UC's open access policies. Let us know how this access is important for you.