Large animal models for stem and progenitor cell analysis.

Extrapolation of an understanding regarding hematopoiesis and, in particular, hematopoietic stem cells (HSCs) from rodent models or in vitro human cell models to applications in humans has proven very difficult. This is not surprising, given the differences between rodent and human hematopoietic physiology and the lack of true in vitro assays for HSCs. Therefore, translational preclinical development of genetic and cellular therapies is dependent on the utilization of practical and well-defined large animal models. This chapter will introduce the most commonly used model species, including macaques, baboons, dogs, cats, and sheep, and explain the particular advantages and limitations of each. Specific protocols for the support of macaques through ablative cell and gene therapy procedures will be included to introduce investigators to the types of resources and support required to maintain a large animal facility dedicated to high-intensity experimentation, and also to introduce investigators to the types of procedures that are possible.