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 same time. We used this technology to look at the bone marrow stem cells from patients with thalassaemia and sickle cell disease to try and work out why it is difficult to correct the stem cells from these patients. This was recently published in Blood (https://doi.org/10.1182/blood.2019002301).

 We found that the common way of counting stem cells doesn't work properly in these patients. This means that these patients are potentially getting much smaller doses of stem cells than clinicians are intending to give. This may explain why there have been problems in the early gene correction trials and it leads the way for better treatments in the future.

 Blood, Cells, Red, Medical, Medicine, Anatomy, Health

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