Small molecules that dissolve stress granules

In ALS, a mutated nuclear protein, FUS (fused in sarcoma), mislocalises to the cytoplasm and aggregates in stress granules. This leads to a liquid to solid transition of the granules and a consequent degeneration of the motor neuron (motor neuron die-back ). We show that lipoamide and lipoic acid can prevent the aggregation of FUS and consequently the die-back of patient-derived motor neurons in cell culture. It also reduced protein aggregation in C. elegans and induced the recovery of motor defects in transgenic D. melanogaster expressing a mutant FUS.

This study takes advantage of the recent advances in understanding liquid-liquid phase separation in health and disease. Read more.

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