IN UTERO MODEL TO ASSESS FATE OF TRANSPLANTED CELLS FOR PEDIATRIC THERAPIES

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 研究表明，早期治疗被诊断患有遗传性疾病的婴儿提供了最好的生存和临床结果，并且未成熟细胞来源可能是移植最有效的。干细胞领域的进展一直受到难以找到允许人体细胞体内分析的预测模型的阻碍。这些研究将探索来自两种不同来源的移植人类细胞的命运。他们将通过解决与临床移植的人类多能细胞相关的基本问题，并为人类进行原理证明转化研究，从而克服瓶颈。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Studies suggest that early treatment of infants diagnosed with an inherited disease provides the best survival and clinical outcomes, and that immature cell sources may be most efficient for transplant. Progress in the stem cell field has been hampered by difficulties in finding predictive models that permit in vivo analysis of human cells. These studies will explore the fate of transplanted human cells from two differing sources. They will have important outcomes towards overcoming bottlenecks by addressing essential issues related to human pluripotent cells for clinical transplantation and conduct proof-of-principle translational studies for humans.