CORE--ANIMAL MODELS

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The Animal Models Core provides critical support for the animal research proposed in all 4 projects. Support is provided for the maintenance of the transgenic mouse colonies used in the Program Project Grant. These colonies provide necessary models for the study of erythropoiesis, hemoglobin switching, and for vector-induced insertional mutagenesis. This Core provides funds for cage costs, and a research technician for genotype and breeding services. This Core also provides the expertise and resources necessary for the rhesus macaque autologous transplantation model used in projects 3 and 4. This model is essential for the further development of safe and effective gene therapy approaches to sickle cell disease. For example, the rhesus macaque model provides an accurate assessment of the efficiency by which lentiviral vectors can transduce human hematopoietic stem cells (HSCs), and whether expression of the transferred globin gene will occur at potentially therapeutic levels. Primates are also necessary to develop in vivo HSC selection strategies, given our prior experience that mouse models alone are insufficient for these studies. The Core provides expert technical services for the entire transplantation procedure, the supplies necessary to accomplish the transplant and the support the animals will require through the period of immunosuppression. We have developed a state of the art primate facility, with particular emphasis on the humane care and psychological well being of the animals, and have the necessary expertise to perform these experiments at a level commensurate with human stem cell transplantation.