In Utero Hematopoietic Stem Cell Transplant for Fanconi Anemia
L. Swartzrock, C. Dib, M. Denis, H. Willner, K. Ho, E. Haslett, M. R. Krampf, A. Girsen, Y. J. Blumenfeld, Y. Y. El-Sayed, M. G. Roncarolo, T. C. MacKenzie, A. 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