Genome editing technology enables study of how leukemia develops in a whole way.  Novel model shows the step-by-step progression of normal blood cells to leukemia and its precursor diseases. The new model could help create replicas of the various stages of leukemia and assist with the evaluation of the efficacy of therapeutic interventions at each stage, according to a study published in Cell Stem Cell.1

Scientists used a genome editing technology called Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) to convert blood cells from patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) to pluripotent stem cells that can mimic all stages of disease progression, from a healthy state to pre-malignancy and finally full-blown leukemia.  

This research marked the first time scientists have been able to transplant leukemia from humans to a test tube and then into mice for study. Researchers at the Icahn School of Medicine at Mount Sinai in New York, New York, report that this new model will empower investigation into the cellular and molecular events underlying the development of leukemia in ways that were not possible before.  Researchers report this new progression model could also be used to develop models for more complex cancers, including solid tumors.

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1. Kotini AG, Chang CJ, Chow A, et al. Stage-specific human induced pluripotent stem cells map the progression of myeloid transformation to transplantable leukemia. Cell Stem Cell. 2017 Feb 16. doi: 10.1016/j.stem.2017.01.009 [Epub ahead of print]