North Carolina-based biotechnology company Chaperone Therapeutics and the nonprofit biotechnology company, the ALS Therapy Development Institute (ALS TDI), have entered into a research partnership to develop therapies for ALS that target protein misfolding and aggregation. Chaperone Therapeutics has identified small molecules that dissolve aggregates of misfolded proteins, a characteristic of many neurodegenerative diseases. One of the targets of these lead compounds is heat shock factor protein 1 (HSF1), a transcription factor that regulates activity of the chaperone proteins that assist with protein folding in the cell (see Feb 2010 news). ALS TDI is conducting preclinical experiments to test the ability of these lead compounds to reverse protein misfolding in cell-based assays and to ameliorate disease in vivo ALS models.
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