Gene Therapy for SCID

The Facts:

Severe combined immune deficiency has been one of the most successfully treated disease by gene therapy. It is caused by a mutation on a single gene, meaning that it is a relatively simple disease to treat. Moreover, it affects bone marrow cells which are easily removed from the body, treated, and put back into the body to carry out their normal function.

Typically, people who suffer from severe combined immune deficiency have to live in a sterilised bubble because they have a non-existent immune system, meaning that normally simple infections such as cold sores or chickenpox could be life threatening. This is why it is often referred to as the boy in the bubble syndrome. Unfortunately, children suffering from this rare disease would require extremely high levels of . antibiotics in order to fight off any infections, and in most cases, people would die before becoming adults.

Gene Therapy Success!:

That was mainly true until now, when gene therapy appears to have made a significant breakthrough in the treatment of this disease. The breakthrough was made by French scientists working on two infants aged 8 months and 11 months old. Bone marrow was taken from each of the patients and grown outside their bodies. This marrow was then infected with a virus carrying the normal gene, before it was transplanted back into the patients. This new gene unblocks the development of other immune cells, and the function of the immune system is restored to normal. The reason that gene therapy for SCID may prove so successful is that the new cells are favoured when they multiply, and so they become relatively high in number in a short period of time youngsters have experienced amazing . It would now appear that the improvements in their diseases, and they no longer live in protective isolation and have normal growth and development.

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Conclusions:

Such findings are very encouraging for the whole field of gene therapy, and there are other stories of success in the treatment of this disease. It is now hoped that the condition will be curable, though further studies and long term analysis of the already treated patients is necessary before the full extent of the success of this therapy can be determined.