De Novo-Designed APC/C Inhibitors Provide a Rationale for Targeting RING-Type E3 Ubiquitin Ligases.
Research output: Contribution to journal › Research article › Contributed › peer-review
Contributors
Abstract
The ubiquitin system represents an attractive pharmacological target for numerous pathological processes, including cancer and neurodegeneration. RING domain-containing E3 ubiquitin ligases constitute the largest class of ubiquitin enzymes, providing a scaffold for substrate recognition and catalysis. Their shallow groove recognition interfaces involving discontinuous epitopes and a lack of defined binding pockets have largely rendered them undruggable. Inspired by natural RING inhibitors, we have developed a pharmacophore-based strategy for the rational design of peptidomimetics targeting RING domains, and we demonstrate its feasibility by using the macromolecular APC/C complex (anaphase-promoting complex/cyclosome). We designed scaffolds binding to the APC/C RING domain and efficiently inhibiting its activity in vitro. Iterative structure-based design and experimental studies to optimize their chemical stability, permeability, and specificity lead to new hydrocarbon-stapled-based molecules inhibiting APC/C in vitro and in cancer cells. Our results provide a robust rationale for targeting RING-containing enzymes of therapeutic value and promising leads for clinical APC/C inhibition.
Details
| Original language | English |
|---|---|
| Pages (from-to) | 11468–11483 |
| Number of pages | 16 |
| Journal | Journal of medicinal chemistry |
| Volume | 68 |
| Issue number | 11 |
| Early online date | 21 May 2025 |
| Publication status | Published - 12 Jun 2025 |
| Peer-reviewed | Yes |
External IDs
| ORCID | /0000-0002-5175-9311/work/184884922 |
|---|---|
| unpaywall | 10.1021/acs.jmedchem.5c00416 |
| Scopus | 105005800473 |