Human Gene Therapy

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Human Gene Therapy Image what it would be like if doctors could cure Huntington's disease, muscular dystrophy, or even hemophilia. Could this be possible? With gene therapy this all could be reality in the near future. Gene therapy is a potential approach to the treatment of genetic disorders in humans. This is a technique where the absent or faulty gene is replaced by a working gene, so the body can make the correct enzyme or protein and consequently eliminate the root cause of the disease (BIO, 1990). There are two types of gene therapy treatment: Somatic cell gene therapy and germline therapy. Somatic cell gene therapy involves obtaining blood cells from a person with a genetic disease and then introducing a normal gene into the defective cell (Coutts, 1998). This type of gene therapy does not prevent the disease from occurring in the next generation because it does not affect the sperm and egg cells. Somatic cell gene therapy only effects the other body cells. Somatic cell gene therapy has to be done several times over the coarse of the patient's life because the effects do not last very long. The contrast of somatic cell gene therapy is germline therapy. Germline therapy takes place in the reproductive cells. It involves the genetic modification of germ cells that will pass the change on to the next generation (Wilson, 1997). This type of gene therapy only has to be done one time to be permanent. One type of germline therapy is to treat a pre-embryo that carries a serious genetic defect before it is placed back in the mother by in vitro fertilization. Another germline therapy is to treat adult sperm and egg cells so the genetic defect is not passed on to children (Coutts, 1998). If a genetic change occurs, it will ... ... middle of paper ... ...ed age where they will be able to understand the effects of the therapy, they should then be allowed to make their own decision. There are many thoughts that have to go into this procedure, and I think people should be careful when considering a life altering gene therapy. References 1. Biotechnology Industry Organization, 1990. Gene Therapy An - Overview. Obtained from TheWWW.10/16/99: http://www.accessexcellence.org/AB/IWT/Gene_Therapy_Overview.html 2. Coutts, M.C., Human Gene Therapy. Obtained from the WWW. 10/16/99: http://www.georgetown.edu/research/nrcbl/scopenotes/sn24.htm 3. PBS On-Line. What is Gene Therapy. Obtained from the WWW. 10/16/99: http://www.pbs.org/wnet/innovation/show1/html/2sb-therapy.html 4. Wilson, J. IHGT The Institute for Human Gene Therapy. Obtained from the WWW. 10/16/99: http://www.med.upenn.edu/ihgt/info/whatisgt.html

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