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Article

VEXAS syndrome.

Blood - 1 Jul 2021

Grayson Peter C, Patel Bhavisha A, Young Neal S

Abstract excerpt

VEXAS syndrome (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) is a monogenic disease of adulthood caused by somatic mutations in UBA1 in hematopoietic progenitor cells. Patients develop inflammatory and hematologic symptoms. Myeloid-driven autoinflammation and progressive bone marrow failure lead to substantial morbidity and mortality. Effective medical treatments need to be identified. Reports in the...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 33971000
DOI: 10.1182/blood.2021011455
PMCID: PMC8462403

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