Sickle cell anemia one more genetic disease cured by gene therapy

Before the gene therapy, Sickle cell Anemia didn’t have any possible cure and the only respite for those afflicted was a blood transfusion. The therapy is most effective when done on affected kids or infants as the older one grows more complications and greater organ damage ensures.

Few days back we had pointed out how Gene Therapy which is essentially the replacement of a malfunctioning gene with a normal well functioning one was the cure of the future. This procedure potentially has the capacity to eradicate all genetic diseases which includes Hemophilia and certain types of cancer. The only problem is it’s not as simple as it sounds.

Gene Therapy has already cured deadly immunity hampering disease ADA-SCID amongst kids and now it has also managed to cure the genetically inherited Sickle Cell Anemia.

So first and foremost what is a Sickle Cell: Sickle’s that instrument which we all hold whenever we visit a farm to pretend how much we know about farming. Sickle Cell anemia is that anomaly which happens when some of the blood cells which should ideally be round like the zeros, morph into a Sickle shaped cell. This causes the Sickle cells to lock together and block the flow of blood to cause severe organ pain and can also be fatal. As of 2103 there were 3.2 million cases of Sickle Cell disease worldwide.

To cure it the doctors went the usual gene therapy way. They removed the patient’s bone marrow, which is the factory producing blood in the body. Made alterations to it, that is shook up the management and fired the unions by adding a virus to the marrow and put it back. The operation was successful, the signs of the disease haven’t cropped up since 15 months in the patient who was 13 years old when the operation took place.

The Sickle Cell Anemia is mainly caused by a fault in a hemoglobin Beta gene found in chromosome 11. Or to explain in simple language, the body’s screws up because of a typo in the instructions to make hemoglobin. Apparently there isn’t any auto correct when it comes to genetics. As the disease is genetic, it’s inherited only when both the parents pass over the faulty hemoglobin gene. If only one of the parents passes the defective gene then the child may become a carrier of the disease. Strangely this disease mostly affects people of African origin.

Also the biggest advantage the gene therapy brings to the table is it doesn’t require any external donor. Hence patients would no longer need to suck up to relatives they barely know for a bone marrow donation. And if they indeed get one then they risk having to bear the nagging reminder of their relative’s favor for the rest of their lives which is almost equally if not more excruciating than the disease they got cured of.

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Categories: Other

Tags: ADA-SCID, Gene Therapy, Genetic Disorders, Hemoglobin, Hemophilia

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