Blood stem cells in sickle cell disease and thalassaemia It looks like it will be possible to cure many inherited genetic disorders like sickle cell disease and thalassaemia within the next decade. It is possible to cure these diseases using gene therapy and genome editing technology which allow defective genes to be corrected in the bone marrow stem cells. These stem cells mature into all the different blood cell types. Correcting a small number of these stem cells allows normal blood cells can be produced indefinitely. This is hugely powerful technology but one of the problems that has been highlighted in the early studies is that these patients stem cells take a long time to regrow after the have been corrected. We looked into this problem in the lab using an amazingly powerful new technology called single cell sequencing. This allow you to work out which genes are active in individual cells. Amazingly, you can use the techonology to look at tens of thousands of cells at the sa