The predisposition to bone marrow failure, cancers, and morphological deformities in Fanconi anemia starts in utero. The first study will test if delivery of normal blood forming cells helps correct the blood problem of the FA fetus after birth. The second will test whether precisely correcting the single DNA mistake that causes FA in the FA fetus can correct blood and other problems found in FA after birth.
Researchers: Agnieszka Czechowicz