In Utero Hematopoietic Stem Cell Transplant for Fanconi Anemia
Leah Swartzrock, Carla Dib, Morgane Denis, Hana Willner, Katie Ho, Ethan Haslett, Mark R. Krampf, Anna Iida Girsen, Yair J. Blumenfeld, Yasser Yehia El-Sayed, Maria Grazia Roncarolo, Tippi C. MacKenzie, Agnieszka D. Czechowicz
This study demonstrates that in utero hematopoietic stem cell transplantation (HSCT) without conditioning can effectively engraft donor cells in FA mouse models, achieving high donor chimerism and potentially offering a safe prenatal treatment for FA.
bioRxiv · May 9, 2024 · PreprintCell Biology