• The Proteostasis Network
• Proteostasis Regulators
• Selected Publications

Proteostasis Regulators

Advances in our ability to characterize and pharmacologically control the Proteostasis Network create new opportunities to ameliorate a wide range of diseases. It is now possible to use orally available small molecules, or Proteostasis Regulators, to restore the natural balance of the Proteostasis Network. It has been demonstrated that Proteostasis Regulators can fold mutated proteins, potentially offering a significant benefit to patients suffering from genetic diseases associated with loss of protein function. Scientists have also shown that Proteostasis Regulators can control other biological pathways within the Proteostasis Network to ameliorate degenerative diseases associated with protein aggregation, such as Alzheimer's and Huntington's diseases.

Proteostasis Regulators function by manipulating one or more signaling pathways to rebalance the Proteostasis Network. Proteostasis Regulators provide a unique and novel therapeutic approach to ameliorate diseases associated with loss of cell function due to accumulation, degradation, or aggregation of misfolded proteins. They also provide a broader approach to genetic diseases than protein replacement therapy, providing access to many cell types, organelles and tissues where recombinant proteins cannot be delivered. It is also a broader approach to correcting genetic diseases than the one-pharmacologic-chaperone:one-disease paradigm of pharmacologic chaperones because a single class of Proteostasis Regulators has the potential to treat multiple related diseases.