Extra Credit: Gene Therapy

Gene therapy is basically inserting a “good” gene where the one before was defective.

a common risk is when foreign “good” gene is inserted, it affects more cells than it is supposed to and could cause cancer. The gene could also be put in the wrong place or become over-expressed. 

There are 2 forms of gene therapy: somatic and germline.In somatic gene therapy some of the patient's genes may be altered to treat a disease, it doesn’t alter the chance that the disease will be passed on to the patient's children.

In germline gene therapy, genes are modified inside the egg or sperm by injecting DNA fragments. Although it might be able to prevent inherited disease, germline gene therapy is controversial and has not been researched very much due to ethical reasons.

Benefits: There are many benefits to people affected by genetic disorders or diseases today. Whether the diseases is widespread and common or rare and deadly, gene therapy can be the cure to many people's daily problems. An article in the New York Times stated that the developer of an eye treatment, Spark Therapeutics, said the treatment had allowed people with certain inherited retinal dystrophies to more easily maneuver in dimmer light than they could before. The company said it planned to apply to the Food and Drug Administration next year for approval to sell the product. 

Although eye treatments are not the biggest change in the world of biotechnology, gene therapy has the potential to cure other more prominent disorders. Cystic fibrosis is a genetic disorder that makes the mucus in the body thick and sticky, clogging airways. An improved gene therapy experiment worked by curing mice with cystic fibrosis and testing cells from the gut of CF patients. Both in mice with cystic fibrosis and in gut cell cultures, this approach yielded positive results. Professor Zeger Debyser from KU Leuven, Belgium said, "We administered the rAAV to the mice via their airways. Most of the CF mice recovered. In the patient-derived cell cultures, chloride and fluid transport were restored... Developing a treatment based on gene therapy will take years of work. For one thing, our study did not involve actual human beings, only mice and patient-derived cell cultures." 

Risks: The viruses that are used to deliver the good genes can affect more than the cells for which they're intended. If a gene is added to DNA, it could be put in the wrong place, which could potentially cause cancer. Genes also can be over-expressed, meaning they can ramp up the production of so much of a protein that they can be harmful by producing the wrong amount of proteins. 

I believe that the benefits do outweigh the risks because these risks can be fixed over time when more knowledge about DNA sequencing. To cure genetic diseases, scientists must first determine which gene or set of genes causes each disease. The Human Genome Project have completed the initial work of sequencing and mapping, but they are becoming more detailed and accurate. This research will provide new strategies to diagnose, treat, cure, and possibly prevent human diseases with gene therapy with fewer errors. Although the risks are dangerous, the idea that many genetic disorders could be cured and even prevented for generations to come is worth the odds of small mistakes. 

Work Cited: 

-Gene therapy: Promising candidate for cystic fibrosis treatment ( http://www.sciencedaily.com/releases/2015/11/151116084010.htm )

-Eye Treatment Closes In on Being First Gene Therapy Approved in U.S.( http://www.nytimes.com/2015/10/05/science/eye-treatment-closes-in-on-being-first-gene-therapy-approved-in-us.html?_r=0 )