Science Advances · 2020 · 298 citations · 49 references
Life SciencesBiochemistryMolecular BiologyBiomedical OpticsBiophotonicsCell SignalingEnzyme FunctionPhotochemistryProtein DegradationBifunctional MoleculesE3 Ubiquitin Ligase Substrate RecognitionMolecular TargetingPhotosensitizersPrecisionPrecision TherapeuticsTemporal Precision
PROTACs (PROteolysis TArgeting Chimeras) are bifunctional molecules that target proteins for ubiquitylation by an E3 ligase complex and subsequent degradation by the proteasome. They have emerged as powerful tools to control the levels of specific cellular proteins. We now introduce photoswitchable PROTACs that can be activated with the spatiotemporal precision that light provides. These trifunctional molecules, which we named PHOTACs (PHOtochemically TArgeting Chimeras), consist of a ligand for an E3 ligase, a photoswitch, and a ligand for a protein of interest. We demonstrate this concept by using PHOTACs that target either BET family proteins (BRD2,3,4) or FKBP12. Our lead compounds display little or no activity in the dark but can be reversibly activated with different wavelengths of light. Our modular approach provides a method for the optical control of protein levels with photopharmacology and could lead to new types of precision therapeutics that avoid undesired systemic toxicity.
49
BET Bromodomain Inhibition as a Therapeutic Strategy to Target c-Myc
Jake Delmore, Ghayas C. Issa, Madeleine E. Lemieux et al. · Cell · 2011 · 2.8K citations · Full text
Kathleen M. Sakamoto, Kyung B. Kim, Akiko Kumagai et al. · Proceedings of the National Academy of Sciences · 2001 · 2.5K citations · Full text
Phthalimide conjugation as a strategy for in vivo target protein degradation
Georg E. Winter, Dennis L. Buckley, Joshiawa Paulk et al. · Science · 2015 · 1.7K citations
Catalytic in vivo protein knockdown by small-molecule PROTACs
Daniel P. Bondeson, Alina Mares, Ian E. Smith et al. · Nature Chemical Biology · 2015 · 1.3K citations · Full text