Rxivist logo

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease with few avenues for treatment. Many proteins implicated in ALS associate with stress granules, which are examples of liquid-like compartments formed by phase separation. Aberrant phase transition of stress granules has been implicated in disease, suggesting that modulation of phase transitions could be a possible therapeutic route. Here, we combine cell-based and protein-based screens to show that lipoamide, and its related compound lipoic acid, reduce the propensity of stress granule proteins to aggregate in vitro . More significantly, they also prevented aggregation of proteins over the life time of Caenorhabditis elegans . Observations that they prevent dieback of ALS patient-derived (FUS mutant) motor neuron axons in culture and recover motor defects in Drosophila melanogaster expressing FUS mutants suggest plausibility as effective therapeutics. Our results suggest that altering phase behaviour of stress granule proteins in the cytoplasm could be a novel route to treat ALS.

Download data

  • Downloaded 4,444 times
  • Download rankings, all-time:
    • Site-wide: 846 out of 83,873
    • In cell biology: 26 out of 4,265
  • Year to date:
    • Site-wide: 983 out of 83,873
  • Since beginning of last month:
    • Site-wide: 1,440 out of 83,873

Altmetric data


Downloads over time

Distribution of downloads per paper, site-wide


PanLingua

Sign up for the Rxivist weekly newsletter! (Click here for more details.)


News