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Gene-addition therapy: A potential cure for sickle-cell disease

The genetic disorder affects millions of Africans, but it could take years befre they benefit.

120 million people worldwide have sickle cell disease — and 65 percent of them are in Africa. It’s the biggest genetic root of death for children. And causes terrible pain for those living with it. The only current treatment — is pain management.

“For me, it feels like my body is starving for oxygen and it’s suffocating. You know what I mean? I feel myself dying,” a sickle cell patient shares.

But now — it appears he has become one of the first people to be cured as part of an American trial.

The disease changes red blodd cells to a sickle shape — that clogs up veins and arteries. The new treatment is called gene-addition therapy.

A gene which creates healthy blood cells is placed inside a harmless version of the HIV virus and then given in a bone marrow transplant.

Directos of National Institutes of Health, Franzis Collins said, “The gene therapy that has now been applied to a small number of patients as part of a clinical trial seems to have worked spectacularly. These patients who had sickle cell disease, now when you look at their blood counts and their blood smears, it looks like they don’t have it anymore.”

The treatment also requires chemotherapy and months of clinical supervision which makes it far too expensive and toxic to be rolled out across Africa for the time being. But researchers say the basic idea could be turned into an affordable cure although that’s likely to take many years.

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