Gene Therapy to Prevent Disease

Gene therapy is an innovative approach to treating and preventing disease (for example: cancer, viral infections, etc.), by replacing a mutated gene with a healthy copy of that same gene. The mere concept of gene therapy was first introduced to the scientific community in the early 1960’s and 1970’s, however it is still in its infancy1 as a medical practice. This technique is experimental and is not yet widely practiced yet in modern medicine because its efficacy is still being thoroughly worked on and investigated.

In the 1960’s and 1970’s, gene therapy was just a concept; an idea fostered by science fiction. However, in 1972, Theodore Friedmann and Richard Roblin published a paper proposing the idea that “ ‘good DNA’ could be used to replace defective DNA in people with genetic disorders”1. This approach to treating diseases was investigated and studied, and administered for the first time on a toddler (a four-year-old child) with a severe congenital immune deficiency (ADA deficiency) on September 14, 1990 by W. French Anderson and his colleagues.2 The patient’s disease, adenosine deaminase deficiency (ADA deficiency), is a congenital or inherited disorder that damages the immune system, leaves it vulnerable to the acquisition of other diseases, and eventually develops into severe combined immunodeficiency (SCID) which causes the individual to lack virtually all immune protection from bacteria or viruses. To help this patient, Anderson extracted white blood cells from the patient’s bone marrow and reinserted the normal genes which produce adenosine deaminase into her system.3 The procedure was successful, and as a result, 300 more clinical gene therapy trials were performed over the next ten years.2

Gene therapy has evolved ...

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...ene Therapy and Germ Line Gene Therapy. Retrieved from http://www.iptv.org/exploremore/ge/features/somatic.cfm

6: Williamson, R., & Kampmann, B. (1993). Retrieved from http://ec.europa.eu/research/biomed/therapy.pdf

7: University of Utah (2004). Exploring Gene Therapy. Retrieved from gslc.genetics.utah.edu/units/genetherapy

8: Bushelle, E., Hertz, E., Hubbard, D., Saylor, K., & Slyker, A. (2003). Technology of Gene Therapy:. Retrieved from http://www.macalester.edu/~montgomery/GeneTherapy.htm

9: Balan, S. (2011). Types Of Gene Therapy - Advantages And Disadvantages of Gene Therapy. Retrieved from http://www.biotecharticles.com/Biotech-Research-Article/Types-Of-Gene-Therapy-Advantages-And-Disadvantages-of-Gene-Therapy-1030.html

10: University of Utah (2014). Gene Therapy Successes. Retrieved from http://learn.genetics.utah.edu/content/genetherapy/gtsuccess/