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Gene Therapy

Gene Therapy

Author: Amanda Soderlind

This lesson will introduce the concept of gene therapy and examine how it can be used by the medical industry to treat various diseases and disorders.

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Source: Video and Images Created by Amanda Soderlind

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Welcome to this lesson today on gene therapy.

Today we will be discussing the process and uses of gene therapy. Gene therapy is basically a process that's used to help fix genetic diseases. As of right now, it's not widely spread because it's still very experimental and costly, and there are some possible dangerous side effects. In testings that have been done, people have died from these different types of testings with gene therapy. But it does give hope to people who have certain genetic diseases that one day gene therapy will be able to fix these diseases.

So it's an application of biotechnology in which mutated genes are replaced with normal genes that code for functional proteins, or genes are inserted that can restore the normal controls over gene activity.

With these genetic diseases, genes are not coding for functional proteins, and it results in this genetic disease. But if these mutated genes could be replaced, or if genes can be inserted that kind of reverse that genetic disorder, it would help people with these different types of diseases.

There are a couple different ways in which genes can be inserted into a person in the process of gene therapy.

Transformation basically uses cells grown in a lab, and it exposes them to DNA with the specified gene-- with a certain gene-- and then it uses an electric current in order to integrate the DNA into this lab grown cell, in order to replace that mutated gene.

Transfection is another way in which genes can be inserted into a cell. In this type we're using a virus. So what happens is that a gene is inserted into a virus, and then the virus will be inserted into the person. And it will transfer that gene to a target cell, and integrate the DNA with the host's DNA.

So these are two ways in which gene therapy can be used to help either replace abnormal genes or restore those normal controls over gene activity.

There have been some different trials with gene therapy. One is with a disease called Severe Combined Immune Deficiency. Severe Combined Immune Deficiency is a type of disorder in which a person's immune system doesn't work, and so basically they have to live in a bubble. Kids with this disorder are often referred to as "bubble kids," because they literally have to live in a bubble and can't be exposed to any germs, because they have a deficient immune system. Gene therapy has actually come a long way with this type of disease, and helping to allow those children to lead a more normal life.

Cystic fibrosis is another example of a disease that has had some trials with gene therapy. Cystic fibrosis, with this disorder, basically they have used the virus that I talked about earlier to deliver normal copies of a gene to the respiratory system. So with cystic fibrosis, you get a buildup of mucus in the respiratory system. By using this virus, they can deliver normal copies of the gene to the respiratory system to help with this condition.

But I think the most common use of gene therapy thus far has been with cancer. It's had the biggest success thus far with gene therapy of any of the other diseases that they've done trials with.

So again, it's not widely spread. This is not something that is commonly used right now because it is still very experimental, very costly, and still can be dangerous, but it is giving people with these genetic conditions or these genetic diseases a lot of hope for the future.

This lesson has been an overview on gene therapy.

Terms to Know
Gene Therapy

The process of replacing mutated genes with normally functioning genes. Gene therapy can be done directly or with vectors (virus).


A gene is inserted into a vector (virus) and injected into a person; the virus will deliver the gene into the host cells.


Using cells in a lab that are grown in certain environments and exposing them to certain DNA in order to express a specific gene.