Gene Therapy As you near the end of the term you will put ev

Gene Therapy As you near the end of the term, you will put everything together you have learned and discuss an important application of genetics: gene therapy. The overall goal of gene therapy is to restore normal function in cells that are affected by genetic disorders. •How does gene therapy work? •What are the challenges of gene therapy? •There are several ethical issues surrounding gene therapy. Pick one and state whether you agree or disagree with the position. Explain your answer. •There are both highs and lows in gene therapy. Research the stories of Ashanti de Silva and Jesse Gelsinger. Based on the outcomes of these treatments, do you think experimental gene therapy trials should continue? Why or why not?

Solution

a). How does Gene Therapy work?

In gene therapy, a normal gene is cloned in place of the mutant gene, responsible for the disease. Replacement of the mutant gene with normal gene results in the synthesis of active protein and the normal function is restored.

b). What are the challenges of gene therapy?

The treatment is short-lived and needs to be repeated at regular intervals to prevent the disease.

The immune system needs to be suppressed each time the therapy is given because the cloning of the gene involves infection with a recombinant virus, The infection of the virus elicits an immune response.

Possible risk of toxicity is associated with the viral infections.

Therapy of multigenic traits is quite complicated

c). There are several ethical issues surrounding gene therapy. Pick one and state whether you agree or disagree with the position.

Gene therapy is a costly affair. All may not afford to use the therapy due to economic reasons. The technique, once available for use must be accessible to all equally irrespective of the economic status.Americal medical Association\'s ethical guidelines consider this as an important point. I agree with the position due to the reason stated above.

d). do you think experimental gene therapy trials should continue? Why or why not?

Yes, I think the experimental gene therapy trials should continue. Ashanti de Silva was cured of the disease, although the effect was temporary but it does show us the light and warrant more and more research to make the therapy long-lived and fool proof. On the other hand, Jesse Gelsinger\'s death discourage us but warrants strict adherence to the ethical guidelines.