De Novo-Designed APC/C Inhibitors Provide a Rationale for Targeting RING-Type E3 Ubiquitin Ligases.
Publikation: Beitrag in Fachzeitschrift › Forschungsartikel › Beigetragen › Begutachtung
Beitragende
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
| Originalsprache | Englisch |
|---|---|
| Seiten (von - bis) | 11468–11483 |
| Seitenumfang | 16 |
| Fachzeitschrift | Journal of medicinal chemistry |
| Jahrgang | 68 |
| Ausgabenummer | 11 |
| Frühes Online-Datum | 21 Mai 2025 |
| Publikationsstatus | Veröffentlicht - 12 Juni 2025 |
| Peer-Review-Status | Ja |
Externe IDs
| ORCID | /0000-0002-5175-9311/work/184884922 |
|---|---|
| unpaywall | 10.1021/acs.jmedchem.5c00416 |
| Scopus | 105005800473 |