Therapeutic modulation of eIF2α phosphorylation rescues TDP-43 toxicity in amyotrophic lateral sclerosis disease models
Therapeutic modulation of eIF2α phosphorylation rescues TDP-43 toxicity in amyotrophic lateral sclerosis disease models
Amyotrophic lateral sclerosis (ALS) is a fatal, late-onset neurodegenerative disease primarily affecting motor neurons. A unifying feature of many proteins associated with ALS, including TDP-43 and ataxin-2, is that they localize to stress granules. Unexpectedly, we found that genes that modulate stress granules are strong modifiers of TDP-43 toxicity in Saccharomyces cerevisiae and Drosophila melanogaster. eIF2α phosphorylation is upregulated by TDP-43 toxicity in flies, and TDP-43 interacts with a central stress granule component, polyA-binding protein (PABP). In human ALS spinal cord neurons, PABP accumulates abnormally, suggesting that prolonged stress granule dysfunction may contribute to pathogenesis. We investigated the efficacy of a small molecule inhibitor of eIF2α phosphorylation in ALS models. Treatment with this inhibitor mitigated TDP-43 toxicity in flies and mammalian neurons. These findings indicate that the dysfunction induced by prolonged stress granule formation might contribute directly to ALS and that compounds that mitigate this process may represent a novel therapeutic approach.
- Department of Genetics Stanford University School of Medicine United States
- UNIVERSITY OF PENNSYLVANIA
- Stanford University United States
- Department of Biology University of Pennsylvania United States
- Department of Biology United States
570, /dk/atira/pure/subjectarea/asjc/1300/1311, Indoles, name=Genetics, Eukaryotic Initiation Factor-2, Immunoblotting, 610, Nerve Tissue Proteins, Poly(A)-Binding Proteins, Article, Animals, Humans, Phosphorylation, Motor Neurons, Analysis of Variance, Adenine, Amyotrophic Lateral Sclerosis, Immunohistochemistry, High-Throughput Screening Assays, DNA-Binding Proteins, Luminescent Proteins, Drosophila melanogaster, Gene Ontology, Ataxins, RNA Interference
570, /dk/atira/pure/subjectarea/asjc/1300/1311, Indoles, name=Genetics, Eukaryotic Initiation Factor-2, Immunoblotting, 610, Nerve Tissue Proteins, Poly(A)-Binding Proteins, Article, Animals, Humans, Phosphorylation, Motor Neurons, Analysis of Variance, Adenine, Amyotrophic Lateral Sclerosis, Immunohistochemistry, High-Throughput Screening Assays, DNA-Binding Proteins, Luminescent Proteins, Drosophila melanogaster, Gene Ontology, Ataxins, RNA Interference
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