Johan Flygare

Our blood consists largely of red blood cells, which are essential for transporting oxygen around the body. Millions of people worldwide have too few red blood cells, a condition known as anaemia. Unfortunately, many patients with anaemia do not benefit from the treatments currently available.

The first aim of the research project is to understand the instructive signal that causes a cell to begin producing red blood cells. The study has already deciphered a substantial part of this code and demonstrated that the signal works by directly reprogramming connective tissue cells from the skin into cells capable of producing red blood cells. By mapping the mechanisms underlying this discovery, we are developing new ways to study and treat anaemia.

The second aim is to identify new therapeutic targets for anaemia by improving our understanding of the factors that stimulate and inhibit the formation of cells that produce red blood cells. Using genetic screening methods, 200 such factors will be studied. In addition to increasing our understanding of how red blood cell production is regulated, the study is expected to identify potential new targets for drug development.

The third aim is to develop new drugs for patients with a specific form of anaemia known as Diamond-Blackfan anaemia. The study has already tested more than 12,000 different compounds and identified a group of molecules capable of correcting the underlying defect responsible for the disease. We now aim to understand how these drug candidates work and refine them so that they can ultimately be used to treat patients with Diamond-Blackfan anaemia.