Gene Therapy Usage in Treating Adenosine Deaminase (ADA) Deficiency

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Gene therapy is a method of correction treatment for damaged gene for inherited disease sufferer. This method first used in treatment for lacks of the immune response genetic disease (genetic immunodeficiency) which is known as lacking of Adenosine Deaminase (ADA) or ADA(-). This ADA enzyme is needed to complete the formation and development of body immune system.

ADA(-) disease attacks new born baby and causes death. The baby is not able to produce antibody system and complete immunization. They easily seize diseases such tetanus, diphtheria, breathing infection disease, measles, and pneumonia. Some patients might have no antibody and immune system at all. This is known as severe combined immunodeficiency – SCID. Usually, children who have disease ADA(-) SCID experience recurring virus, fungus, and bacteria infection and have high risk of early cancer infection.

The best treatment method for this disease is gene therapy. With this method normal ADA gene from donor is admitted into bone marrow which is the major blood cell producer organ. But this method involves bone marrow separation which is very hard to be done. Other easier technique is to use T lymphocyte cell. Lymphocyte or white blood cell is responsible to prevent diseases and also fight cancer cell. Normal ADA gene from T lymphocyte cell is separated and transplanted into human cell through retroviral vector; an amended gene of a virus.

Illustration explaining how gene therapy is done to treat ADA Deficiency
Gene Therapy for ADA Deficiency

Part of the virus gene is replaced with normal human ADA gene which is needed to be transferred. The viral-vector is then mixed and bred together with normal human cells in laboratory. The vector later enters the normal cells then forming new genes and stays permanently inside the chromosome. The new gene is known as virus-mediated gene.

The transfer of the new virus-mediated gene into the T lymphocyte cell of ADA(-)SCID ‘s patient is done by intravenous injection. Therefore ADA(-) gene is now repairable so that it can returns to produce normal ADA enzyme. The repaired gene of T lymphocyte cells can live normally in the receiver’s body. In fact it lives longer than the unrepaired T lymphocytes. Cell implant of the repaired gene should be done continuously once in a few months. Instead, if the gene therapy can be done to bone marrow stem cell, ADA(-)SCID children may be fully cured with only one treatment without the continuous need of new ADA gene implant.

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