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.
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.
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.