Sickle cell disease cured by gene knock-out

Switching off a single gene can help treat sickle cell disease by keeping the blood forever young. The illness is caused by a mutant form of adult haemoglobin, but not by foetal haemoglobin. Targeting BCL11A, the gene responsible for the body’s switch-over from foetal to adult haemoglobin, effectively eliminates the condition in mice.

The mutant form of adult haemoglobin forms long sticky chains inside red blood cells. The cells containing these chains can clog small blood vessels, depriving organs of oxygen and causing pain. In severe cases, sickle cell disease can be fatal. Tricking the body into make foetal haemoglobin again can alleviate symptoms, though.

That’s because foetal haemoglobin does not form sticky chains. However, it is produced in the body only during development in the womb and in the six months following birth. It has a higher affinity for oxygen than adult haemoglobin, vital in allowing the developing foetus to steal oxygen from its mother’s blood — via redwolf.newsvine.com

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