what is gene therapy illustrate using the example of adenosine deaminase deficiency

Dear Student, 
  • 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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