ARTICLE | Targets & Mechanisms

Heat shock and awe

January 19, 2012 8:00 AM UTC

U.S. researchers have identified several small molecules that decrease toxic protein aggregation in vitro.1 The first-generation compounds have been licensed by Proteostasis Therapeutics Inc. and are proof of concept for the biotech's approach to treating protein misfolding diseases like Huntington's and cystic fibrosis.

Cells have evolved a complex network of quality control enzymes to prevent the formation and accumulation of misfolded proteins during normal cellular function. However, this protein homeostasis (or proteostasis) network often is overwhelmed in protein misfolding diseases, which generate high levels of protein aggregates that are impossible to clear and lead to cell death...