Year 3

The current treatment for many childhood blood-related diseases is complicated by toxic regimens that are needed to ensure transplanted cells from donors persist and do not result in complications that can compromise health. These studies focus on new ways to safely transplant the necessary blood stem cells to treat these diseases prior to birth, and new techniques to monitor the fate of the cells post-transplantation. Outcomes of these studies have shown efficient methods to expand umbilical cord blood hematopoietic stem cells needed in the quantity required for efficient transplantation, safe and clinically relevant methods to transplant the cells prenatally, and effective methods to monitor the cells postnatally that confirm persistence and in the anatomical sites where they reside.