Sickle cell anemia and beta-thalassemia are two of the more common hereditary blood disorders, causing scientists to try to find ways to predict and treat these conditions before they evolve to unmanageable levels.
Sickle cell anemia causes the hemoglobin protein in red blood cells to take on a "sickle" shape, unlike normal cells that are round and smooth, while beta-thalassemia involves a hemoglobin deficiency. Both are genetic conditions caused by different genes, and each have their own unique set of related health problems, including poor blood flow, organ damage and anemia.
A new treatment developed by Weill Cornell Medical College researchers could provide relief to these patients. The treatment involves transferring a normal beta-globin gene, via a patient bone marrow transplant, into red blood cells that have become deformed by each disease. After the procedure, the transplanted cells are expected to produce normal beta-globin and hemoglobin.
While the treatment has only been tested on one patient, who suffered from beta-thalassemia, doctors think it will also work for those with sickle cell anemia. They are cautiously optimistic, however, as the challenge for that particular condition is to add healthy genes without multiplying both normal and deformed cells. The therapy may be more effective for beta-thalassemia patients, as their hemoglobin cells are deficient, not deformed.
"The variable nature of the beta-thalassemia mutations suggests that some patients would be better candidates for gene therapy than others, and that success of gene therapy depends on the ability of a specific vector to make hemoglobin," lead researcher Dr. Stefano Rivella told Science Daily. "This is something we can test in advance using a little bit of a patient's blood – which is quite extraordinary."
Techniques such as this gene therapy treatment could revolutionize medical treatments going forward and produce better health outcomes for patients. As diagnostic laboratories refine techniques, they may need to hire proven researchers who are devoted to the tenets of personalized medicine and genomics.
This article is brought to you by Slone Partners, a leading laboratory recruitment firm in the emerging sciences of molecular, clinical, and in-vitro diagnostics, anatomic pathology and personalized medicine.
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