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How gene therapy is done to treat ADA de...

How gene therapy is done to treat ADA deficiency?

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During gene therapy the lymphocytes from the blood of the patient are removed and grown in a nutrient culture medium. A healthy and functional human gene, ADA cDNA encoding enzyme is introduced into the lymphocytes using a retrovirus. The genetically engineered lymphocytes are subsequently returned to the patient. Since these cells are not immortal, the patient requires periodic infusion of such genetically engineered lymphocytes. The disease could be cured permanently if the gene for ADA isolated from bone marrow cells are introduced into the cells of the early embryonic stages.
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