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  Gene therapy

Gene therapy:

The actuality that genes can be cloned to several thousand copies via genetic engineering has given mount to a completely novel model of therapeutic device namely, Gene therapy. Gene therapy includes the substitution of corrective genes in place of imperfect genes in human. There are two kinds of gene therapy. They are:

1). Somatic cell gene therapy and
2). Germ line cell gene therapy.

Both might be used for treating the inherited diseases. In somatic cell gene therapy the patient’s cells are obtained from blood or bone marrow, or skin and brain, treated via genetic engineering and then substituted to the patient. Somatic cells are non-reproductive cells. Such cells and their genes whenever corrected, the genetic modifications will not be inherited to their children. On other hand, this kind of treatment could cure diseases caused by single gene mutations. In this therapy, retroviruses are used as carriers of the genes. Their genetic material, RNA is copied into DNA. To generate effective vectors certain necessary genes are spliced out from the viruses. This will render the virus not dangerous and permit space for the therapeutic genes to be inserted. Hundreds of millions of copies of the genes are required for somatic cell gene therapy. To take them, the vectors must also be multiplied in equivalent numbers. Experimentally, these enormous copies of virus vectors are made employing helper virus. The integrated vector and the cloned gene are employed in somatic cell gene therapy. Somatic cell gene therapy has been successful in animals, until now.

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