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Phase 1 study of quercetin, a natural antioxidant for children and young adults with Fanconi anemia

16 Jan 2025

Abstract excerpt

ABSTRACT: Fanconi anemia (FA) is a rare inherited disorder characterized by progressive bone marrow failure (BMF) and a predisposition to malignancy. Systemic reactive oxygen species (ROS) and increased sensitivity of FA hematopoietic progenitors to ROS play a key role in the pathogenesis of BMF. Treatment with antioxidants improve hematopoietic function in Fancc-/- mice. We report the safety, tolerability, and...

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PMID: 39820512
DOI: 10.1182/bloodadvances.2024015053

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