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Gene Therapy

Gene Therapy

Description:

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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Tutorial

This lesson is going to give an overview of gene therapy by looking at:

  1. Gene Therapy
  2. Uses

1. Gene Therapy

Gene therapy is a process that's used to help fix genetic diseases. As of right now, its use is not widely spread because it's still very experimental and costly. There are also some possible dangerous side effects. People have died from these different types of testing with gene therapy, but it does give hope to people who have certain genetic diseases that one day gene therapy will help.

Term to Know

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

It's an application of biotechnology, and there are a couple of ways it can be used. Mutated genes can be replaced with normal genes that code for functional proteins, or genes are inserted that can restore the normal controls over gene activity. With genetic diseases, genes are not coding for functional proteins.

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

  • Transformation - uses cells grown in a lab and it exposes them to DNA with a specified gene. Then it uses an electric current in order to integrate the DNA into this lab grown cell to replace that mutated gene.
  • Transfection - is a way in which genes can be inserted into a cell using a virus. A gene is inserted into a virus, and then the virus will be inserted into the person. It will transfer that gene to a target cell, and integrate the DNA with the host's DNA.

Terms to Know

  • Transformation
  • Using cells in a lab that are grown in certain environments and exposing them to certain DNA in order to express a specific gene.
  • Transfection
  • A gene is inserted into a vector (virus) and injected into a person; the virus will deliver the gene into the host cells.


2. Uses

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; they have to live in a bubble.

Children 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. With this disorder, scientists have used a virus to deliver normal copies of a gene to the respiratory system. 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.

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.

Summary

Gene therapy is still experimental and costly. This is the process of replacing mutated genes with normally functioning one. Transformation and transfection are two ways in which genes can be inserted into a person. Gene therapy could have several uses and much potential. Some of the most promising include Severe Combined Immune Deficiency or Cystic Fibrosis. Research with cancer looks the most promising.

Keep up the learning and have a great day!

Source: This work is adapted from Sophia Author Amanda Soderlind

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).

  • Transformation

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

  • Transfection

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