Targeted Protein Degradation by Small Molecules

Annu Rev Pharmacol Toxicol. 2017 Jan 6:57:107-123. doi: 10.1146/annurev-pharmtox-010715-103507. Epub 2016 Oct 12.

Abstract

Protein homeostasis networks are highly regulated systems responsible for maintaining the health and productivity of cells. Whereas therapeutics have been developed to disrupt protein homeostasis, more recently identified techniques have been used to repurpose homeostatic networks to effect degradation of disease-relevant proteins. Here, we review recent advances in the use of small molecules to degrade proteins in a selective manner. First, we highlight all-small-molecule techniques with direct clinical application. Second, we describe techniques that may find broader acceptance in the biomedical research community that require little or no synthetic chemistry. In addition to serving as innovative research tools, these new approaches to control intracellular protein levels offer the potential to develop novel therapeutics targeting proteins that are not currently pharmaceutically vulnerable.

Keywords: IMiDs; PROTACs; chemical knockdown; protein degradation; ubiquitin proteasome system.

Publication types

  • Review
  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Drug Delivery Systems / methods*
  • Drug Delivery Systems / trends
  • Humans
  • Oligopeptides / administration & dosage
  • Oligopeptides / metabolism
  • Proteins / antagonists & inhibitors
  • Proteins / metabolism*
  • Proteolysis / drug effects*
  • Small Molecule Libraries / administration & dosage*
  • Small Molecule Libraries / metabolism*

Substances

  • Oligopeptides
  • Proteins
  • Small Molecule Libraries