Using iPSCs to unravel hereditary anemias: from fundamental knowledge towards novel therapies

Project summary

Diseases of red blood cells
In some congenital diseases, making healthy red blood cells is a problem. Standard treatments are blood transfusions or bone marrow transplants, but suitable donors are scarce.

Deploy IPSCs
We will use patient iPSCs to: 1) study disease mechanisms, 2) create patient-specific blood-forming stem cells, and 3) develop better treatments. We will investigate whether the new treatments are available, acceptable, accessible and affordable for patients, clinicians and policy makers.

New treatments
We want to make the blood-forming stem cells suitable for individual patients, or to make cultured blood for many patients. Because rejection reactions are prevented in this way, a suitable donor is no longer necessary. Thus, these can become curative treatments for patients with hereditary anemia.