Using patient-derived iPSCs to develop humanized mouse models for chronic myelomonocytic leukemia and therapeutic drug identification, including liposomal clodronate.

Induced pluripotent stem cells (iPSCs) can generate any mature (differentiated) cell type. By generating iPSCs from patients, we can generate large numbers of relevant cells by re-differentiation for disease modeling and drug screening. Here, we generate and re-differentiate iPSCs from a patient with chronic myelomonocytic leukemia (CMML), a disease of the blood. Using this novel system, we identify a potential new drug treatment for CMML.