Gene Therapy

By: Bella Seitz

What is gene therapy and how is it effective?

What is Gene Therapy?

Gene therapy is a way that people can get rid of, or cure, a certain disease.


  • Gene therapy is a treatment
  • Gene Therapy is done by adding a correct copy of a defected gene

    Its is a treatment that alters your genes inside your bodies cells to stop the disease in your body.

    The DNA in your body codes for the control of your body’s form and function

    • This could be like making you grow taller

    • But, the genes in your body that don’t work the way they should, can cause disease

    Gene therapy takes a bad gene and replaces it with a new one to try and cure the disease.

    it can also improve the ability your body has to fight a disease.

What diseases are cured by gene therapy?

Some diseases that can be treated by gene therapy include:


  • Cancer
  • Cystic Fibrosis
  • Heart disease
  • Diabetes
  • Haemophilia

Why Gene Therapy is done

Examples are in the text

Gene therapy is mainly used to fix bad genes to treat a genetic disease, or to help you fight them better.

Researchers are trying to find many ways that they can do this, such as replacing a mutated gene.

According to Mayo Clinic, "Some cells become diseased because certain genes work incorrectly or no longer work at all." The website also says that if malfunctioning genes that are not working are replaced, the disease can be treated.


This can also be done by fixing the mutated gene, which could be like turning off a bad gene so it doesn't cause the disease. Another way a disease can be treated is by turning on a good gene to help cure the disease, or making the diseased cell more evident to the immune system. This means that the doctors can train your genes to recognize that these genes are a threat.

What are the risks of gene therapy?

“Gene therapy has some potential risks. A gene can't easily be inserted directly into your cells. Rather, it usually has to be delivered using a carrier, called a vector. The most common gene therapy vectors are viruses because they can recognize certain cells and carry genetic material into the cells' genes.” says Mayo clinic.

Some risks may include:

  1. An unwanted immune system reaction- Which can make the new gene seem like a threat and could even lead to organ failure

  2. Targeting the wrong cells- Which means not just one cell could be targeted it could spread to others and lead to the healthy genes going bad

  3. Infection caused by the virus-It could occur that once recovered to the body the virus may be able to cause that disease

  4. Possibility of causing a tumor-If new gene are put into the wrong spot into your DNA this could lead to a tumor formation

How gene therapy is done

Gene therapy can be done in 2 ways; Outside of your body, or ex vivo, and inside of your body, or in vivo. Ex vivo treatment takes out the malfunctioning cells of the body and puts new healthy genes in them and places them back into the body. In vivo treatment requires that the good gene is put directly into a tumor, which can be hard depending on where it is.
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Examples of gene therapy that partially cured patients with blindness

Researchers from perelman school of medicine at the university of pennsylvania and the children's hospital of philadelphia did a study on that focused on gene therapy for partially treating blindness.
They were able to improve eyesight in 3 adult patients each had before been treated in one eye.They did the same treatment in the second eye.
The patients were able to see in low-light places or rooms. And could also find their way around. The patients are now able to walk around at night, go shopping, and recognize people's faces.
There were no bad effects that happened after.
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How they choose people for gene therapy

If you are even going to even consider it, your answer must be yes, to “Could the condition be corrected by adding one or a few functional genes?” Says the University Of Utah.
You need to know what gene you want to fix. And also have a copy of your DNA in your lab. You need to know all you can about how the gene factors into the disorder that you may have.

Works cited

"What Is Gene Therapy?" What Is Gene Therapy? N.p., 2015. Web. 22 Oct. 2015. University of utah


"Gene Therapy Proves Effective in Treating Blindness." Medical News Today. MediLexicon International, 9 Feb. 2012. Web. 24 Nov. 2015.

Medical news today


"Moores Cancer Center | UC San Diego Health System." UC San Diego Health System. UC San Diego, 2015. Web. 24 Nov. 2015.

Cancer UCSD



"Gene Therapy." - Mayo Clinic. Mayo Clinic, 5 Jan. 2013. Web. 24 Nov. 2015.

Mayo Clinic