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In utero hematopoietic stem cell transplantation for Fanconi anemia

11 Jul 2024

Abstract excerpt

Fanconi anemia (FA) is a grievous inherited DNA repair disorder resulting in the inability to repair interstrand crosslinks. 1,2FA is caused by mutations in 1 of 23 different FA complementation (FANC) genes, with the most frequent mutations occurring in FANCA (~60%-70%), FANCC (~10%), and FANCG (~10%). 3The increased chromosomal fragility in FA individuals leads to progressive bone marrow failure (BMF) and a...

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PMID: 38991119
DOI: 10.1182/bloodadvances.2023011894

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