Curing sickle cell anemia with a patient's own stem cells

This is great news for people with sickle cell anemia!  Sickle cell is a genetic disorder where a person does not make normal hemoglobin.  Instead, a single base mutation in their DNA makes them produce hemoglobin-S, a protein that causes their red blood cells to "sickle."  These sickled red blood cells interfere with blood flow through small blood vessels, depriving tissues of oxygen. This causes pain, anemia, infections, organ damage, and stroke. 

In the past, normal stem cells from umbilical cord blood for example could create normal hemoglobin for those with sickle cell, but donor compatibility was always a problem.  Researchers have now found away to make a patient's own stem cells create normal hemoglobin.  Here is how they do it:

To prevent the production of the abnormal hemoglobin that causes sickle cell disease, a viral vector was introduced in cell cultures of patients who have the disease. The vector carried a therapeutic globin gene harboring an embedded small interfering RNA precursor designed to suppress abnormal hemoglobin formation. Tested in adult stem cells from SCD patients, researchers found that the newly formed red blood cells made normal hemoglobin and suppressed production of the sickle shaped hemoglobin typical of the disease.

For those that need a translation this means they were able to genetically cure sickle cell.  They used an engineered virus to deliver both an inhibitor to the abnormal hemoglobin gene and then a normal hemboglobin gene to a culture of cells from patients with sickle cell.  After treatment, these "cured" stem cells produced normal hemoglobin.  In theory, these cells could then be injected back into the patient without fear of rejection like with donor stem cells.  Michel Sadelain, MD, PhD, explains:

Sickle cell disease can only be cured by transplanting healthy blood-forming stem cells from another individual, but this option is not available to most patients due to the difficulty in finding a compatible donor.  By using gene transfer, there is always a donor match because the patient’s own stem cells are used to treat the disease.

No need for cloning folks!  Once again your own stem cells are all that may be needed.