Gene therapy

Gene therapy

The process of replacement of faulty gene with a normal healthy gene, to treat diseases where other medical approaches are not effective, is called gene therapy. The first successful attempt on gene therapy was done on September 14, 1990 by French Anderson and his team (National Institute of Health US) to treat a four - year old girl, born with a rare genetic disease called Severe Combined Immuno Deficiency (SCID).

SCID is caused by a defect in the gene for the enzyme adenosine deaminase (ADA). The persons suffering from SCID have defective immune system, because their lymphocytes are functionless. Gene therapy involves the extraction of lymphocytes from the bone marrow of the sufferer. Then by using vector (retrovirus), a good copy of human gene encoding the enzyme (ADA) is introduced into the lymphocyte. Now the vector contains the ADA gene but no viral genes. The bone marrow stem cells removed from the SCID patient are co - infected with the vector and helps virus to produce new virus particles with ADA genes. This equips these cells with a good copy of the gene in addition to the existing faulty gene. The treated cells are re-Injected to the patient's bone marrow. These cells can play their normal role in patient's immune system.

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