Sickle cell disease gene therapy

A teenager in France has had his sickle cell disease reversed due to treatment changing his DNA.

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Sickle cell disease is a condition that affect the shape of  red blood cells so that they are unable to carry out there function of transporting oxygen throughout the body as well. The cells are normally round in shape but the condition makes them rod shaped. This is caused by a inherited faulty gene that affects the development of the red blood cells. Not only can the cells not transport as much oxygen but they also have a chance to block up blood vessels that in extreme cases can lead to a stroke or lung problems.

Some people don’t have the disease but are carriers so contain the potential for the children to have the condition rather than themselves. This can have implications wheather to have children as they could be a carrier of the condition or actually have it themselves which could have a impact on there quality of life. This makes it important for suffers to have there partners checked so a decision can be reached.

To combat this there a variety of treatments that are available depending on the severity of the disease and the individual. This can be due to regular painkillers such as ibuprofen and paracetamol or in some cases stronger in a hospital. Antibiotics can be used to counteract the higher risk of infection from the disease as well as ensuring that a person is fully vaccinated. A medicine called hydroxycarbamide can be used to reduce symptoms. Regular blood transfusions is used in extreme cases where the condition gets worse or when it damages parts of the body.

A newly explored treatment involves changing the genetic instructions in bone marrow, the part in bones that makes red blood cells, to rectify the coding error to make healthy red blood cells. A teenager in France has had this treatment done by using a virus to infect the bone marrow with the correct coding for them. The person used to have to go in for blood transfusions every month to dilute his blood but now 15 mouths after treatment they aren’t on any medication and appear to be completely rid of the disease. This example shows how gene therapy can give someone there lives back and can potentially improve the quality of lives for millions of people. The treatment needs to be tested on more people to see if it is viable as a mainstream treatment. The treatment can only be given in state of the art hospitals and laboratories so it will still be a while before it is widely accessible particularly due to the majority of suffers being in Africa. Even so this shows potential to play a massive role in treating sickle cell disease.

http://www.bbc.co.uk/news/amp/39142971

http://www.nhs.uk/Conditions/Sickle-cell-anaemia/Pages/Introduction.aspx#treatment

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Thanks for reading

George

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