A sterol-binding pocket in iRhom1 underlies paralog-specific regulation of the sheddase ADAM17
ADAM17, the major sheddase in mammalian cells, releases membrane-tethered EGFR ligands and inflammatory cytokines, and is a central regulator of cell signalling. The rhomboid pseudoproteases, iRhom1 and iRhom2, function as essential cofactors of ADAM17, controlling its maturation and activation. In contrast to the well-characterized iRhom2, the mechanism and regulation of its ubiquitously expressed paralog iRhom1 remain undefined. Here, we present a 2.5 angstrom cryo-EM structure of the full-length human iRhom1/ADAM17 complex, revealing a previously unrecognized sterol-binding pocket located between TMD2 and TMD5. Structure-guided mutagenesis and pharmacological perturbation of sterol binding demonstrate that sterol binding is required to stabilize the iRhom1/ADAM17 complex and sustain its shedding activity. Strikingly, this regulation is paralog-specific: iRhom2 precludes sterol binding and instead stabilizes ADAM17 through direct intramolecular interactions. Furthermore, two human iRhom1 variants associated with cardiac disease localize adjacent to the sterol-binding pocket and disrupt ADAM17 maturation and activity. Together, these findings uncover mechanistic divergence between iRhom paralogs and establish a sterol-binding pocket in iRhom1 as a critical determinant of ADAM17 stability, revealing a potential avenue for paralog-selective therapeutic targeting.
Authors
- Fangfang Lu (ORCID: https://orcid.org/0000-0002-9214-3046)
- Chia‐Hsueh Lee (ORCID: https://orcid.org/0000-0003-0819-6477)
- Matthew Freeman (ORCID: https://orcid.org/0000-0003-0410-5451)
- Hongtu Zhao
- Yaxin Dai
Institutions
- Broad Institute (US)
- St. Jude Children's Research Hospital (US)
- University of Oxford (GB)
- Guangzhou Experimental Station (CN)
Publication Details
- Journal
- bioRxiv (Cold Spring Harbor Laboratory)
- Published
- 2026-05-01
- DOI
- https://doi.org/10.64898/2026.04.28.721421
- Primary Topic
- HER2/EGFR in Cancer Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00