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Gene therapy targets sickle-cell disease


Elliott Vichinsky estimates that at least 30% of his adult patients with sickle-cell disease die from preventable causes. Red blood cells are supposed to be shaped like concave discs, but in people with sickle-cell disease, a mutation in a single gene collapses them into a crescent shape. The pointy sickles catch on each other and clog blood vessels. They cut off oxygen to limbs. They cause kidney failure, hypertension, lung problems and strokes — along with bouts of excruciating pain.

These are common and treatable complications, so why the high death rate? Vichinsky attributes it to a lack of infrastructure, such as care centres, to properly monitor adults with sickle-cell disease. This is partly because the disease mainly affects low-income minorities and people in developing countries. “If they were tracked before,” says Vichinsky, “they would not be dead.”

 

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