Beating Severe Combined Immunodeficiency Syndrome (SCID)

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Imagine living your entire life contained within a plastic bubble. Contact from the outside world, including your parents, is lethal. Rather than feeling the warm touch of a human hand, the clammy cold of laboratory gloves comforts you to sleep. Is this living or this surviving? You make the call. SCIDs is an acronym for Severe Combined Immunodeficiency Syndrome. Persons born with SCIDs lack the ability to fight off infections. SCIDs creates a situation in which the common cold is just as deadly as pneumonia. This family of diseases is obviously debilitating and life-threatening. That is why finding a cure is imperative. This disease is not contagious, it is genetic and is thus acquired through the simple role of the genetic dice. There is absolutely nothing we can do about this sad syndrome, or is there... We can look to gene therapy: an exciting and revolutionary new field of research and medicine which may reveal the key to unlocking a myriad of genetic diseases. This paper will explore the problems posed by SCIDs and the answer offered by Marina Cavazzana-Calvo and Salima Hacein-Bey. Their work in gene therapy has great potential towards bursting the bubble on SCIDs. Immune System Introduction So, you ask, what does it mean to have SCID? Well, in order to answer this question, we have to go through a quick tour of the immune system. The immune system of the human body is comprised of a vast array of cells that fight off diseases (antigens) that are harmful to the well-being of the body. In an individual with a properly functioning immune system, the body has multiple genes that encode specific instructions for the proper design and function of the cells of the immune system. T... ... middle of paper ... ...g gene. Thus, the non-functional gamma-c chain of these patients was corrected to function like a normal gamma-c chain. The researchers then took the corrected cells and returned them to the patients. Did it work? Yes. The results show a dramatic increase in a variety of major immune system cells. Where are they now? Both patients left protective isolation after approximately three months of treatment. After an 11 month analysis, both patients appear to be growing well. No side effects (of any kind) have been reported. Basically, both patients have achieved a nearly perfect recovery in less than a year! On a third patient, similar positive results have been achieved after 4 months of treatment. Drs. Cavazzana-Calvo et. al. have BURSTED THE BUBBLE ON SCIDs!!! Of course, long-term analysis must be completed in order to see the duration of this treatment.

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