Understanding gene therapy starts with understanding what genes do.
In this video, a part of our GT-FAQ video series, Nicki, a scientist at our Genetic Therapies Center of Excellence, shares a quick refresher on how genes help the body make proteins and what happens when a gene isn’t working properly. She also shares how gene therapy is intended to work by delivering a new, working copy of a gene so the body can make the protein it needs. Watch for a clear, simple explanation of the basics.
Read the video transcript below.
Video Transcript
To understand gene therapy, we need a brief biology refresher – specifically, let’s start with our DNA.
What is a gene?
A gene is a short piece of DNA that tells the cell how to make a specific protein. Proteins do lots of important jobs in the body. In people who have a genetic disease, certain genes aren’t working properly, so the proteins they make don’t work the way they should.
What is gene therapy?
Gene therapy, sometimes called gene transfer therapy, is a way to deliver a new functioning copy of a gene that helps your body make a protein.
How does it work?
I like to use the example of getting a new toy or getting a new electronic device. Many times these toys already come with batteries inside. But sometimes the batteries don't work.
So what do you do in that situation? You don't fix the battery that's in the toy. You actually put a new battery in.
In gene therapy, we’re not doing anything specifically to change the genes in your body. We're simply delivering a new version of the gene to make a protein.
Let’s review!
- Genes are pieces of DNA that tell cells how to make proteins.
- Genetic diseases can happen when a gene doesn’t work properly.
- Gene therapy aims to deliver a new, working copy of the gene.
- This helps your body make the protein it needs.
If you have questions about gene therapy, talk to your healthcare provider or email our Patient Affairs team at [email protected].