Gene-therapy introduction

Introducing gene therapy

Gene therapy is a technique that changes genes. It may help manage or treat a genetic disease. If you’re living with a genetic condition, gene therapy may be a possibility now or in the future.

Jump to: What is gene therapy? What is gene therapy? Gene therapy types Gene therapy types Methods Methods

What is gene therapy?

The goal of gene therapy is to target genetic diseases at the root. Gene therapy permanently changes a disease-causing gene by adding a new gene, turning a gene on or off, or editing the specific gene.

These changes affect how the proteins that cause disease function.

Person looking hopeful about gene therapy

Examples of gene therapy

Let’s look at how gene therapy may help people in real life.

  • In some inherited eye diseases, a mutation to a single gene causes problems with vision. For these patients, gene therapy could find and treat the gene that is causing the problem. The goal of this therapy is to improve patients’ vision.
     
  • There are also some blood and skin disorders caused by a mutation to a single gene. Researchers are now studying ways that gene therapy can target those genes to change the course of the disease.

Discover more gene-therapy research

How does gene therapy work?

Gene therapy is a technique. Like any technique it can involve multiple steps.

Magnifying glass over DNA strand for targeting specific genes in gene therapy

FIND

Gene therapy alters a specific gene.

Two strands of DNA with plus symbol icon representing gene addition and pencil icon representing gene editing

CHANGE

A gene can be added, turned on, or turned off inside the cell.

Illustration of person with a pencil icon representing edited genes building proteins

MODIFY

The newly added or altered genes change the proteins.

DNA strand highlighting new proteins working in place of faulty proteins

CREATE

The new proteins may work in place of the faulty proteins.

Common types of gene therapy

Gene therapy possibilities are changing in exciting ways. There are 2 main types of gene therapy: gene addition and gene editing.

You might be wondering how these 2 therapy types differ. The goal is the same: to correct a gene that is not working properly. The main difference is how these 2 therapies accomplish that goal.

Understanding the differences between gene addition and gene editing

When we learned about genetics and mutations, we talked about how your genes are like your body’s instruction manual. When there is a mistake, the body doesn’t work the way it’s supposed to, and a genetic disease results.

Gene therapy works in different ways. To get a better idea, we have to think about different problems you may find in the instruction manual.

Instruction manuals can contain incorrect information. For example, if 1 word is incorrect, it can change the meaning of a sentence. Let’s explore 2 ways you could correct the instructions:

Instruction manual illustration representing errors in the instructions
DNA strand with pencil icon representing gene editing

Gene editing:
correcting the manual

What it does…
Gene editing aims to change your DNA at a targeted location.

What it means…
Updates incorrect information in the instruction manual with a change or edit.

How it works…
Different technologies edit specific genes within the body. The goal is to potentially eliminate or create proteins to treat or manage a genetic disease.

Gene editing:
correcting the manual

What it does…
Gene editing aims to change your DNA at a targeted location.

What it means…
Updates incorrect information in the instruction manual with a change or edit.

How it works…
Different technologies edit specific genes within the body. The goal is to potentially eliminate or create proteins to treat or manage a genetic disease.

DNA strand with plus sign symbol representing gene addition

Gene addition:
adding a new page

What it does…
Gene addition aims to treat a disease by adding a gene when one is not working properly.

What it means…
Corrects the instruction manual by adding a new page.

How it works…
A delivery vehicle, known as a vector, carries the new gene to the intended target. The goal is to help cells create proteins so the body works as intended.

Gene addition:
adding a new page

What it does…
Gene addition aims to treat a disease by adding a gene when one is not working properly.

What it means…
Corrects the instruction manual by adding a new page.

How it works…
A delivery vehicle, known as a vector, carries the new gene to the intended target. The goal is to help cells create proteins so the body works as intended.

Each person’s body is unique and may not respond the same as someone else.

Learn more about gene editing

Learn more about gene addition

Gene-therapy methods

There are 2 ways to deliver gene therapy to address gene mutations: outside the body, known as ex vivo, or inside the body, known as in vivo.

DNA strand with pencil icon representing gene editing

Ex vivo means gene therapy is added to your cells OUTSIDE of your body.
In this process:

  • Certain cells are removed from your body and taken to a lab
  • Scientists make changes to the genes inside of the cells
  • These changes treat or correct mutations
  • Cells are then returned to your body
DNA strand with plus sign symbol representing gene addition

In vivo means that gene therapy is given straight to your cells INSIDE your body.
In this process:

  • Gene therapy is typically administered through an IV
  • Cells are edited inside the body

Both ex vivo and in vivo methods require a delivery vehicle, or vector, to get the treatment into the cell.

Gene therapy isn’t a new idea. The Food and Drug Administration (FDA) has approved several gene therapies, and there are more being researched. The future of medicine is full of possibilities.

You’ve learned about the types and delivery methods of gene therapy. Next, let’s look at the research and how it’s affecting genetic diseases in the real world.

Gene therapy: today and tomorrow

Learn more

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