Cellular Reprogramming toward the Erythroid Lineage

Abstract EN

Haemoglobinopathies such as thalassaemia and sickle cell disease present a major health burden.

Currently, the main forms of treatment for these diseases are packed red blood cell transfusions and the administration of drugs which act to nonspecifically reactivate the production of foetal haemoglobin.

These treatments are ongoing throughout the life of the patient and are associated with a number of risks, such as limitations in available blood for transfusion, infections, iron overload, immune rejection, and side effects associated with the drug treatments.

The field of cellular reprogramming has advanced significantly in the last few years and has recently culminated in the successful production of erythrocytes in culture.

This paper will discuss cellular reprogramming and its potential relevance to the treatment of haemoglobinopathies.