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In Utero Hematopoietic Stem Cell Transplant for Fanconi Anemia

2024-05-14

Abstract excerpt

Fanconi Anemia (FA) is an inherited DNA-repair deficiency caused by mutations in diverse Fanc genes that leads to bone marrow failure and malignancies. FA disease begins at early embryonic stages, and while FA prenatal testing has long been available, no fetal therapies for FA currently exist. Postnatally, FA hematologic disease can be cured through allogeneic hematopoietic stem cell transplantation (HSCT); howev...

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Literature Corpus work
f24c2d30-8a62-5620-9a3d-0a2e34129885
DOI
10.1101/2024.05.09.592452
Open publication

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In Utero Hematopoietic Stem Cell Transplant for Fanconi AnemiaDOI 10.1101/2024.05.09.592452
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