Immune deficient mice (SCID) can be injected with human hematopoietic stem (CD34+) cells, resulting in generation of a human immune systemin a small animal setting (SCID-hu mouse). These mice have a significant advantage over cell culture systems and other larger animal models. Cell culture systems fail to recapitulate biological nuances of an "in vivo" (or whole animal) setting. Larger animal models that are currently used to investigate human viruses suffer in that these agents are not tested in human cells and that large animal models are very expensive. SCID-hu mice provide a quick system to establish human immune cells and this is a dynamic, self-regenerating system. Gene therapy vectors designed to express therapeutic genes in human immune cells can be quickly evaluated in this setting. SCID-hu mice can also be infected with human oncogenic viruses such as KSHV, HTLV, and EBV to decipher early events in leukomogenesis as a result of infection with these viruses. Human malignancies propagated in SCID mice and chemotherapeutic agents against these tumors can be quickly evaluated in an in vivo setting. Finally, embryonic stem cells (ESC's) can be injected into SCID mice to test the differentiation profiles prior to implantation into humans for therapeutic uses. ESC's can quickly and rigorously be evaluated as to their pluripotency and for any deficiencies/tumorigenicity prior to clinical uses.

File Number: R1625-100 

Disease: Cancer

Other Information: Principal Investigator: Gerold Feuer