GENE THERAPY is done to correct gene defect in an individual. In this method normal gene for functioning is introduced into the cell to cure the defect.
The first clinical gene therapy was given in 1990 to a 4 year old girl, with adenosine deaminase (ADA) deficiency. The enzyme is required for immune system to function. The disorder in girl arose is due to deletion of the ADA gene.
For gene therapy, lymphocytes from the blood of the patient are separated and cultured outside the body. These lymphocytes were introduced with cDNA using a retroviral vector. These genetically engineered cells were returned to the patient. These cells are not immortal and a periodic transfer of recombinant cells is required in patients.
If the cDNA is introduced in an egg or early embryo, a permanent cure can be achieved.
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