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I.9 Gene therapy

Gene therapy is defined as the introduction of normal genes in place of defective genes in the cells to correct genetic disorders.

The gene therapy is of two types:

  1. Somatic or non-inheritable gene therapy involving delivery of a normal gene into the individual’s somatic cell. Some of the patient’s own cells are removed, repaired and re-implanted in the patient.
  2. Germ-line or inheritable gene therapy involving delivery of a normal gene into the embryo.

Gene therapy has been employed in diagnosing ADA deficiency, cystic fibrosis, haemophilia, AIDS. Tay-Sach’s disease, asthma, brain tumour, melanoma, cancers of
breast, ovaries, prostate, liver and colon, muscular dystrophy, neurological disorders, diabetes, cardiovascular diseases, etc.

The first clinical gene therapy was given in 1990 to a four-year old girl Ashanthi DeSilva who was suffering from the rare genetic disorder adenosine deaminase-deficient severe combined immunodeficiency disease (ADA-SCID).

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