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Inflammatory cell death and monocyte dysfunction in VEXAS syndrome - PubMed

5 hours ago
  • #inflammatory cell death
  • #VEXAS syndrome
  • #UBA1 mutation
  • VEXAS syndrome is a severe adult-onset autoinflammatory disease caused by somatic mutations in the UBA1 gene.
  • UBA1 mutations disrupt myeloid cell function, leading to TNF-α-induced cell death via RIPK1 phosphorylation, MLKL, and caspase-8 pathways.
  • Defective NF-κB responses and reduced cFLIP(L) expression contribute to aberrant apoptotic and necroptotic cell death in UBA1-mutated monocytes.
  • UBA1-mutated monocytes show blunted cytokine responses to TLR agonists despite preserved TLR expression.
  • UBA1M41V-derived macrophages exhibit a pro-inflammatory profile, increased chemokine secretion, and impaired efferocytosis due to lysosomal dysfunction.
  • The study highlights a pathogenic axis linking UBA1 loss of function to inflammatory cell death, impaired signaling, and defective resolution mechanisms in VEXAS syndrome.