Gene Therapy and Genetic Disorders

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Every cell in the human body is given instruction by our genes as to what job each cell should perform. Every tiny detail is dependent upon the instruction of our genes. If a gene or cell becomes damaged, changes, or is missing, there are major consequences to the body and the health of the individual. Genetic disorders are caused by damaged or missing genes and can be life threatening. They can lead to serious diseases in the individual. Certain types of genetic disorders cannot be cured by treatment with medicine. Drugs may help relieve symptoms of certain disorders but cannot cure the disease. Drugs cannot reverse or repair a damaged cell or gene. Scientists and doctors have done extensive research to find ways to reverse the negative effects of a damaged gene or repair a missing or damaged gene in order to cure diseases. This led to the discovery of gene therapy. Gene therapy’s main focus is on replacing or repairing defective genes within the body in the hope that this will repair the defect in the gene and in doing so cure or at least lessen the negative effects of the disease. The procedure involves the introduction of a healing or working gene copy into specific cells of an individual in order to repair or replace a damaged gene. Two main methods have been tried —adding a gene to substitute for a gene that wasn't working as it is supposed to be, or changing genes that are not working properly. Cystic fibrosis, hemophilia and sickle cell anemia are among the diseases where gene therapy has been considered. Diseases that are incurable in which gene therapy has also been considered include Parkinson’s, HIV/AIDS, Alzheimer’s, diabetes, cancer, and heart disease. ...

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...ause it could be very dangerous since it gives an individual the power to change lives when the benefits or consequences of that power are not completely understood or adequately studied. Genetic gene therapy should be limited to treatment of genetic disorders and must be approached with caution, careful research and strict guidelines.

Works Cited

Gene Therapy – Treating Disease by Repairing Genes – Joseph Panno, PH.D.

http://www.gtherapy.co.uk/

http://www.gtherapy.co.uk/pros-and-cons

http://www.gtherapy.co.uk/what-is-gene-therapy

http://www.gtherapy.co.uk/gene-therapy-ethics

http://www.macalester.edu/~montgomery/GeneTherapy.htm

http://www.bio-itworld.com/2014/1/29/gene-therapys-next-generation.html

http://www.theguardian.com/science/2014/mar/05/hiv-gene-therapy-gm-cells-trial-success

http://www.ndsu.edu/pubweb/~mcclean/plsc431/students98/fleck.htm

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