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Information on SCID

SCID is an acronym which stands for severe combinedimmunodeficiency, and it is a very serious, sometimes life threatening disease.Sometimes it is also referred to as the “boy in the bubble” syndrome. The immunesystem protects the human body from various different types of infections. Twotypes of white blood cells or lymphocytes are present in the immune system andthose are B cells and T cells. B cells are in charge of the humoral immunityand they are responsible for the production of antibodies which act in the bodyfluids. T cells are in charge of the cell-mediated immunity and they are responsiblefor boosting other sorts of immune cells in their efforts to oppose variousharmful substances. When a personsuffers from defected T cells and B cells, the condition is known as the severecombined immunodeficiency disease. It is actually a genetic disorder and thereare two different types of it. XSCID involves a mutated X chromosome which isknown by the name of IL2RG. ADA SCID involves a mutated gene in which is incharge of the encoding of the ADA. ADA is an acronym for Adenosine Deaminase,an important type of enzyme which is responsible for the production of new DNAin the human body. Those who suffer from severe combined immunodeficiencydisease usually suffer from numerous types of infections because their immunesystems do not function properly. 1 out of 80,000 newborns suffers from SCID,and unfortunately they do not survive long after their birth.

What is Gene Therapy?

Gene therapy involves the insertion of genes into thetissues and cells in order to treat various kinds of hereditary and geneticdisorders. It may also be used for the prevention of numerous kinds of geneticdisorders. Basically, it replaces all the defective genes with the new ones.

Gene Therapy for Human Severe Combined ImmunodeficiencyDisease

The gene therapy treatment for those who suffer from severecombined immunodeficiency disease involves the replacement of the mutated genesin the X chromosome or the replacement of the mutated genes which encode ADAwith new, normal genes. Once the replacement is through, the immune systemstarts functioning properly. The procedure took place in 1990 for the firsttime. Gene therapy for those who suffered from XSCID triggered leukemia inseveral cases so it was discontinued. ‘Bubble boy’ and bone marrow transplantare other options for those who suffer from SCID.

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