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.
(1) Genetic manipulations, such as replacing defective or missing genes with healthy ones, can be used to alter germ cells (egg or sperm) and somatic cells. Theoretically germ-line gene therapy appears to have more advantages since it aims at preventing a genetic defect from being transmitted to future generations. However, the prospects of germ-line gene therapy look more remote due to many unresolved ethical and social problems as well as technical obstacles. (2) What is presently understood as gene therapy is, mostly, somatic cell gene therapy. By altering the genetic material of somatic cells onetime cures of devastating, inherited disorders may be potentially achieved.
The chromosome, called human artificial chromosome, is duplicated and passed along just like a normal one (5). The first disease that was approved for gene therapy was ADA deficiency, in which the body lacks production of adenosine deaminase. Adenosine deaminase is important for the proper function of the immune system; people who lack this enzyme have a poor immune response to disease and normally die within the first few years of life (1). The procedure used to correct this disease was to make regular infusions of the patients own genetically corrected T-lymphocytes (1). First the lymphocytes were taken from the patients and encouraged to grow using a disabled mouse retrovirus as a vect... ... middle of paper ... ...o make the perfect child.
Researchers hope that in the coming years, every genetic disease will have gene therapy as its treatment. Gene therapy could be the last therapy that the human race will ever need. What is Gene Therapy? So what is this mystical new wonder called gene therapy? Gene therapy is the introduction of genes into existing cells to prevent or cure a wide range of diseases.
One must also consider the ethical questions that arise. Gene therapy offers undeniable benefits, but the risks it poses need to be addressed before this technology can become common practice. The goal of gene therapy is to correct the unwanted trait or disease by introducing a modified copy into the cell. Notice that the purpose is not to replace defective genes in the host cell, rather it is to provide a new copy, so the correct protein is expressed, or at least the defective gene is neutralized (Blachford 462). Humans are made of trillions of cells, each with a specific function.
While others think that it is a breakthrough and should be used to its full potential. Patients with diseases like Cystic Fibrosis, Parkinson’s disease, and Alzheimer are currently making use of gene therapy to help decrease the outcome of their disease. (1)(3) By altering the genetic makeup of a person, it is giving the offspring chances to possibility not have the disease passed onto them. (1) While some diseases caused by genetic issues are fixed or slowed down, it is possible that doing gene therapy could be creating brand new ones. Anytime a gene is messed with, there is a risk of mutation.
Available from www.nih.gov/news/panelrep.html. Verma, Inder M., and Nikunj Somia. (1997). “Gene therapy – promises, problems and prospects.” Nature 389: 239-242.
The Ultimate Therapy. Tikkun May-June 1998, v1, n3, p33(7) Obtained from WebPals on 10/6/98. Wilson, Jim. Institute for Human Gene Therapy. Last modified: 10/20/97.Obtained form the WWW 9/3/98:http://www.med.upenn.edu /ihgt/info/whatisgt.html