Sickle cell disease

In subjects affected by sickle cell disease (SCD), the red blood cells do not receive enough oxygen. As a result, they become hard and sticky, resembling a sickle. These hard blood cells can clog the vessels and block blood flow to the tissues, causing episodes of severe pain and anemia.

SCD is the most common inherited blood disorder worldwide, transmitted when a child receives a sickle cell gene from both their parents. Millions of people globally suffer from this hereditary blood dysfunction. To date, the only cure for this disease consists of bone marrow or stem cell transplantation. However, of note is that these are heavy procedures that may potentially lead to serious undesirable effects.

New treatments are being investigated, though. In the California Institute for Regenerative Medicine (CIRM), about 23 clinical-stage programs in cell and gene therapy are currently ongoing.

One of these new therapeutic procedures, headed by Doctor Mark Walters, involves the hematopoietic removal of stem cells from the patients’ bone marrow. Therapeutic beta-globin genes are then added, leading to the production of anti-sickling hemoglobin. When introduced back into the patients, these new hematopoietic cells are able to generate healthy blood cells. By this means, the need for a donor and the risk of rejection are eliminated.