Functional Analysis of Human Hematopoietic Stem Cells In Vivo in Humanized Mice.

Ex vivo generation and expansion of functional hematopoietic stem cells represents the holy grail of reprogramming and would constitute a major advance in stem cell therapies and generation of blood cellular products. In vivo testing is critical to assure proper cell intrinsic function in an organismal context. Here we describe methods for the generation of human hematopoiesis chimeric mice and evaluation of hematopoietic stem cell function. The choice of mouse model, stem cell source, and transplantation route can be adjusted to suit the desired application.