Gene therapy is a treatment method that was first proposed by medical researchers in 1972. The idea is to focus on making changes to or inserting new genetic information directly into the chromosomes of human cells to stop a disease on a genetic level.


It wasn’t until 1980 that the first try at gene therapy was attempted by Dr. Martin Cline, a UCLA geneticist. He inserted genes into a patient to treat a blood disorder called beta-thalassemia. The procedure produced no result.


As of today, gene therapy is still in the experimental stage and is not widely available to the public. After more than four decades, medical researchers still have complex problems to solve in this cellular science before a widespread medical treatment becomes ready for use among the public.


The concept of gene therapy is easy to understand, however.


The goal is to find a way to introduce new genetic material into cells to compensate for genes that are malfunctioning and creating diseases. For example, sometimes genes mutate abnormally. Since genes are the “blueprints” by which new cells are manufactured in the body, a mutated cell might start manufacturing cancer cells.


If those cancer-producing genes can be “switched off,” modified or replaced with normally functioning genes, the result could be incredible cures to some of the worst diseases known to humankind.


The current approach to gene therapy does not call for inserting a gene directly into a cell. Rather, a carrier called a vector is engineered to serve as a “delivery vehicle” of new genetic material.


Researchers got this idea from what they know about natural viruses. They use vectors to attach to cells and inject them with their own genetic blueprints. Once the genes of the virus are inside a healthy cell, they take it over and began creating copies of themselves.


Medical researchers are working diligently to produce an artificial virus to serve as a vector for delivering positive genetic material into malfunctioning human cells.


Note that some people who suffer from incurable diseases are already undergoing experimental gene therapy treatments. At this point, however, it is considered a treatment of last resort.


Professor John Rasko of the University of Sydney called gene therapy “a science still in its infancy” but an area of medical research that “holds great promise.”