For years, physician scientists have been trying to model the abnormal blood cell production that occurs in patients with DBA. These efforts have involved studies on yeasts, flies, fish and mice. Human models have been difficult to create and previously relied on samples of patient bone marrow. As published this week in BLOOD, a group of collaborating scientists at The Children’s Hospital of Philadelphia show that skin cells from patients with DBA can be reprogramed so that, instead of skin, they start to produce blood cells (http://www.ncbi.nlm.nih.gov/pubmed/23744582). These reprogrammed cells, termed induced pluripotent stem cells (iPSCs) show defects in blood production that are characteristic of DBA when compared to iPSCs from normal individuals. The work also demonstrates that the abnormal blood cell production is caused by the genetic mutation, because when the reprogramed cells are corrected for the mutation, blood cell production becomes near normal. Thus, for the first time, DBA can be modeled in the culture dish and each model is specific for the patient from whom the skin cells were taken. Moreover, the genetic defect can be corrected in iPSCs, suggesting new approaches for gene therapy. This new technology is likely to accelerate progress in understanding DBA. For example we can compare reprogramed cells from individual patients who have only mild anemia to those from their relatives who are transfusion dependent or we can test new (and old) medications to identify ones that improve blood cell production with less toxicity than steroids. In the longer term blood cells produced in the culture dish may one day be used for patient specific therapy. Although there still is a long way to go, these are exciting times for DBA patients and research. Importantly, this progress would not be possible without the support of DBA patients and families, including their participation in research. (For more information and/or study participation in iPSC research for DBA, contact Weissmi@chop.edu and Besslerm@email.chop.edu).