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Scientists Discover Better Potential Treatment for Sickle Cell Disease

Update Date: Jul 01, 2013 01:07 PM EDT

New research reveals a promising treatment for Sickle cell disease.

Sickle cell disease is an inherited form of anemia.  The autosomal recessive disorder is caused by mutations in hemoglobin that deform red blood cells.  In healthy people, red blood cells are flexible and round and can move easily through blood vessels.  However, in patients with sickle cell anemia, the red blood cells become rigid and sticky and are shaped like sickles or crescent moons.  These deformed cells often get stuck in small blood vessels and slow or block blood flow and oxygen to parts of the body. 

While there is still no cure for the sickle cell disease, treatments can relieve pain and help prevent further problems associated with the disorder.

Researchers note that a small number of patients have been successfully treated with allogeneic hematopoietic stem cell (HSC) transplantation.  However, they say that there are several drawbacks and complications associated with this type of procedure, such as graft vs. host disease and long-term immune suppression.

A new study published recently in the Journal of Clinical Investigation, revealed that many of the complications associated with allogeneic hematopoietic stem cell transplantation could be prevented by the use of autologous HSC transplant, which uses patient cells that have been treated genetically modified to replace defective hemoglobin.

Researchers in the latest study wanted to see if a genetic vector that encodes a human hemoglobin gene can be engineered to block sickle hemoglobin and stock the sickling of red blood cells.  Researchers were able to efficiently transduce bone marrow cells from sickle cell disease patients using the vector.  The transduced cells were then successfully transplanted into immunocompromised mice, suggesting that this method could potentially be used to treat sickle cell disease.  

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