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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.

PDFDOI

02 /Specification & Classification

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