More Into Gene Therapy

Prospects for gene therapy

Several methods are now available for introducing new genetic material into mammalian cells. These methods allow consideration of a more direct approach to treatment of genetic diseases, namely, gene therapy or introduction of a functional gene to replace or supplement the activity of a resident defective gene. Typically, two strategies have been considered, germ-line and somatic cell gene therapy, which differ in the nature of the recipient cells. In the germ-line model, foreign DNA is introduced into the zygote or early embryo with the expectation that the newly introduced material will contribute to the germ line of the recipient, i.e., be passed on to the next generation. By contrast, in somatic gene therapy models, genetic material is introduced only into somatic cells and is not transmitted to the germ cells.

Key ideas:

-       Germ-Line Gene Therapy

-       Somatic Cell Gene Therapy

Source:

Fauci, Anthony S. Harrison's Principles of Internal Medicine, 14th Edition. New York: McGraw-Hill, Health Professions Division, 1998. Print.

Paraphrasing:

Gene therapy is the process of introducing healthy genes into the human body, or other mammalian beings. The treatment mainly focuses on genetic diseases by making a defective mutated gene come in contact with a healthy functional gene as to alter the resulted proteins of the gene that is the main source of the disease.

This treatment has two different approaches, they are known as the germ-line gene therapy and somatic cell gene therapy. The germ-line gene therapy targets gametes cells in the gonad organs. It enables healthy functional genes to be injected into either the zygote or an early embryo as to, hopefully cure the patient, as well as pass it down to their offspring and other future generations. On the other hand, the somatic cell gene therapy targets the other cells of the body. As it does not include sex cells, it has no possibilities of passing down the changes to their children or further generations.