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Discussing 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?
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?

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Solution Preview

Gene therapy is the use of DNA as a drug to treat disease by putting therapeutic DNA directly into the cells of a patient. In short, gene therapy uses genes to treat diseases. There are several ways this is done. The most common include:

1. Replacing a mutated gene that causes disease with a healthy copy of the gene.
2. Inactivating, or "knocking out," a mutated gene that is functioning improperly.
3. Introducing a new gene into the body to help fight a disease

Gene therapy is considered controversial because of the risks associated. There are several challenges to gene therapy. These include:

1. Unwanted immune system reaction. Your body's immune system may see the newly introduced viruses as intruders ...

Solution Summary

An overview of Gene Therapy and the challenges that surround it are provided.