Article

Attitudes Toward Prenatal Interventions in the Fanconi Anemia Community

2025-08-19

Abstract excerpt

<h4>Background</h4> In-utero cell and gene therapies could become effective treatments for many inherited diseases. Notably, recent pre-clinical data supports that in-utero hematopoietic stem cell transplantation ( IU -HSCT) can be curative for bone marrow disease in Fanconi anemia (FA) enabling preventative treatment without use of genotoxic conditioning agents or immune suppression. Given this, we surveyed pati...

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Literature Corpus work: 91307792-1fdf-57b1-bd9d-b7ae89a81027
DOI: 10.1101/2025.08.15.25333795

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