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Complexome profiling showed impaired immunoproteasome assembly in a novel PRAAS subtype caused by monoallelic PSMB8 variants

Wijngaard, R.; van der Made, C. I.; Kalkan Ucar, S.; Ramakrishnan, G.; Rosenfeld, J. A.; Vogel, T. P.; Nicholas, S. K.; Weisz-Hubshman, M.; Undiagnosed Diseases Network, ; Cabrera-Orefice, A.; Siebels, B.; Mair, T.; Schluter, H.; Smeets, R. L.; van Beek, R.; van Karnebeek, C. D. M.; Hoischen, A.; Vissers, L. E. L. M.; Wortmann, S. B.; Wevers, R. A.; Oud, M. M.; Guerrero-Castillo, S.

2025-06-26 genetic and genomic medicine
10.1101/2025.06.25.25330124 medRxiv
Show abstract

Immunoproteasomes, essential for MHC class I antigen presentation, differ from standard proteasomes by incorporating the catalytic subunits PSMB9 ({beta}1i), PSMB10 ({beta}2i), and PSMB8 ({beta}5i). Proteasome-associated autoinflammatory syndromes (PRAAS) are type I interferonopathies resulting from impaired proteasome function. Here, we describe two individuals carrying monoallelic de novo variants in PSMB8, both presenting with early-onset systemic autoinflammation and features of immunodeficiency, accompanied by a marked type I interferon response. To investigate the underlying mechanism, we performed complexome profiling on interferon-{gamma}-stimulated fibroblasts harboring the p.(Ala235Asp) variant. This variant, located at the {beta}-ring interface, disrupted proper assembly of the immunoproteasome, resulting in reduced levels of fully assembled 20S and 26S immunoproteasomes and accumulation of assembly intermediates. The findings suggest a dominant-negative effect and broaden the clinical and genetic spectrum of PRAAS with immunodeficiency (PRAAS-ID), while highlighting the utility of complexome profiling to study proteasome assembly defects.

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