The recent development of induced human pluripotent stem cells (iPS cells) from differentiated adult cells has been heralded as potentially a major advance in medicine. iPS cells can be genetically manipulated and differentiated into a variety of adult tissues. The technology can be used to model disease pathogenesis, to screen for drugs that may be used to treat diseases and, perhaps one day, to produce healthy tissues to replace those affected by disease. Here we discuss these possibilities in the bone marrow failure syndrome Diamond Blackfan Anemia.