what is gene therapy illustrate using the example of adenosine deaminase deficiency
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- Gene therapy involves altering the genes inside your body's cells in an effort to treat or stop disease.
- The new DNA usually contains a functioning gene to correct the effects of a disease-causing mutation.
- Adenosine deaminase deficiency is an inherited disorder that results in severe combined immunodeficiency (SCID)
- It results in severe abnormalities of the immune system and leaves children unable to fight infection.
- Adenosine deaminase (ADA) deficiency was the first human disease treated with gene therapy.
- A retrovirus, which is capable of transferring it's DNA into normal eukaryotic cells (transfection), is engineered to contain the normal human ADA gene.
- Isolated T-cell stem line cells from the patient are exposed to the retrovirus in cell culture, and take up the ADA gene.
- Reimplantation of the transgenic cells into the patient's bone marrow establishes a line of cells with functional ADA, which effectively treats SCID.
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