Question

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Answer 1

Gene therapy is the curing of a genetic disorder by introducing into the individual a normal gene to replace or overcome the effects of a mutant gene.

Two types of gene therapy are possible --

1.Somatic cell therapy- in which somatic cells are modified genetically to prevent a genetic defect in the individual. Somatic cell therapy results in the treatment of genetic disease in the individual, but progeny could still inherit the mutant gene.

2. Germ line cell therapy -- in which germ-line cells are modified to correct a genetic defect . Germ line cell therapy ,however ,could prevent the disease because the mutant gene can be replaced by the normal gene and that normal gene can be inherited by offsprings.

Both the somatic and germ line cell therapy had been used successfully in nonhuman organisms(eg-mice) but only somatic cell therapy had been used in human.

Somatic gene therapy proceeds as follows- a sample of individual's cells carrying the defective gene was taken . Then normal, wild type copies of the mutant gene were introduced into the cells, and the cells were reintroduced into the individual. This the cells will be produced a normal gene product, therefore the symptoms of the genetic disease were completely or partially reversed.