Department of Cancer Biology, Dana-Farber Cancer Institute, Boston, MA, USA; Department of Biological Chemistry and Molecular Pharmacology, Harvard Medical School, Boston, MA, USA.
Department of Biological Chemistry and Molecular Pharmacology, Harvard Medical School, Boston, MA, USA; Department of Chemical and Systems Biology, Chem-H and Stanford Cancer Institute, Stanford School of Medicine, Stanford University, Stanford, CA, USA.
Cell Chem Biol. 2022 Oct 20;29(10):1470-1481.e31. doi: 10.1016/j.chembiol.2022.08.003. Epub 2022 Sep 6.
Targeted protein degradation (TPD) uses small molecules to recruit E3 ubiquitin ligases into the proximity of proteins of interest, inducing ubiquitination-dependent degradation. A major bottleneck in the TPD field is the lack of accessible E3 ligase ligands for developing degraders. To expand the E3 ligase toolbox, we sought to convert the Kelch-like ECH-associated protein 1 (KEAP1) inhibitor KI696 into a recruitment handle for several targets. While we were able to generate KEAP1-recruiting degraders of BET family and murine focal adhesion kinase (FAK), we discovered that the target scope of KEAP1 was narrow, as targets easily degraded using a cereblon (CRBN)-recruiting degrader were refractory to KEAP1-mediated degradation. Linking the KEAP1-binding ligand to a CRBN-binding ligand resulted in a molecule that induced degradation of KEAP1 but not CRBN. In sum, we characterize tool compounds to explore KEAP1-mediated ubiquitination and delineate the challenges of exploiting new E3 ligases for generating bivalent degraders.
靶向蛋白降解(TPD)利用小分子将 E3 泛素连接酶募集到靶蛋白附近,诱导泛素依赖性降解。TPD 领域的一个主要瓶颈是缺乏可用于开发降解剂的可及 E3 连接酶配体。为了扩展 E3 连接酶工具包,我们试图将 Kelch-like ECH-associated protein 1 (KEAP1) 抑制剂 KI696 转化为几种靶标的招募工具。虽然我们能够生成 BET 家族和鼠焦点黏附激酶(FAK)的 KEAP1 招募降解剂,但我们发现 KEAP1 的靶标范围很窄,因为使用 cereblon (CRBN)-招募降解剂很容易降解的靶标对 KEAP1 介导的降解有抗性。将 KEAP1 结合配体与 CRBN 结合配体连接,得到一种既能诱导 KEAP1 降解又不能诱导 CRBN 降解的分子。总之,我们表征了工具化合物以探索 KEAP1 介导的泛素化,并阐明了利用新的 E3 连接酶生成双价降解剂的挑战。