Hematopoietic stem cell gene therapy has become a successful therapeutic strategy for some inherited genetic disorders. Pre-clinical toxicity studies performed to support the human clinical trials using viral-mediated gene transfer and autologous hematopoietic stem and progenitor cell (HSPC) transplantation are complex and the use of mouse models of human diseases makes interpretation of the results challenging. In addition, they rely on the use of conditioning agents that must induce enough myeloablation to allow engraftment of transduced and transplanted HSPC.
View Article and Find Full Text PDFThe Syrian hamster, Mesocricetus auratus, is a suitable rodent species for standard regulatory toxicity studies. However, little is published about the female Syrian hamster reproductive system. It has unique anatomic features that differ from the other rodent species.
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