To understand and integrate the risks and the physiologic benefits, stem cell transplants must be studied in vivo. The primary intent of preclinical studies is to acquire this experience (i.e., conduct safety, feasibility, and efficacy testing). There are numerous questions that should be asked during these preclinical studies, including: Which animal models best mimic the target human popula tion and disease process? Are stem cells stabile and physiologically active following transplantation? What is the best site and developmental stage for stem cell transplantation? Should the transplanted population include both progenitor and terminally differentiated cells? Do tumors form? Do the grafted cells survive in high efficiency? Selection of the most appropriate animal model will depend on the questions being asked as well as the disease state being studied. Here, we have included some considerations for developing a preclinical model to evaluate stem cell therapies to treat certain endocrine diseases.
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