Well Being

Gene therapy research presents hope for sickle cell anemia

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Scientists are used to being cautious. But I was reading this article and I was beside clapping for the science!

See, whenever we get very good results from our experiments, we always tell ourselves "let's test this some more", "let's confirm this in some other population". Well, let's. But the results of this new study are so encouraging that we ought to celebrate with virtual champagne!

Gene therapy has successfully treated sickle cell anemia in mice! OK, so it's in mice but read on first.

The scientists introduced the gene for gamma-globin into the mice's blood-forming cells and then introduced those altered cells into  (sickle-cell anemic) mice. The investigators found that months after they introduced the altered blood-forming cells, the mice continued to produce gamma-globin in their red blood cells.

With gene therapy, the mice were able to produce their own normal gamma-globin. And there's more. Transplanting the altered blood-forming cells from the treated mice into a second-generation sickle cell mice showed that the second generation also corrected their disease!

Again, this is in mouse models, so there's caution in celebration. But it's a potential eh?

Sickle cell anemia is an inherited disease caused by a mutation in a single nucleotide of the B-globin gene that unfortunately changes the resulting amino acid from glutamate to valine. That single change creates abnormal hemoglobin that causes red blood cells to form "sickle-shape". The sickle cells block blood vessels and don't carry enough hemoglobin for the body's requirement, leading to various medical complications.