Gene therapy for human severe combined immunodeficiency disease

Information on SCID

SCID is an acronym which stands for severe combined immunodeficiency, and it is a very serious, sometimes life threatening disease. Sometimes it is also referred to as the “boy in the bubble” syndrome. The immune system protects the human body from various different types of infections. Two types of white blood cells or lymphocytes are present in the immune system and those are B cells and T cells. B cells are in charge of the humoral immunity and they are responsible for the production of antibodies which act in the body fluids. T cells are in charge of the cell-mediated immunity and they are responsible for boosting other sorts of immune cells in their efforts to oppose various harmful substances. When a person suffers from defected T cells and B cells, the condition is known as the severe combined immunodeficiency disease. It is actually a genetic disorder and there are two different types of it. XSCID involves a mutated X chromosome which is known by the name of IL2RG. ADA SCID involves a mutated gene in which is in charge 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 DNA in the human body. Those who suffer from severe combined immunodeficiency disease usually suffer from numerous types of infections because their immune systems 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 the tissues and cells in order to treat various kinds of hereditary and genetic disorders. It may also be used for the prevention of numerous kinds of genetic disorders. 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 severe combined immunodeficiency disease involves the replacement of the mutated genes in the X chromosome or the replacement of the mutated genes which encode ADA with new, normal genes. Once the replacement is through, the immune system starts functioning properly. The procedure took place in 1990 for the first time. Gene therapy for those who suffered from XSCID triggered leukemia in several cases so it was discontinued. ‘Bubble boy’ and bone marrow transplant are other options for those who suffer from SCID.