A common question we hear is whether gene therapy changes your genes. In this video, part of our GT-FAQ video series, Nicki, a scientist at our Genetic Therapies Center of Excellence, walks through the basics of DNA and genes. And she explains how gene therapy works without changing your DNA. Watch for a simple explanation.
Read the video transcript below.
Video Transcript
Does gene therapy change a person’s genes?
The short answer is no.
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.
What is a genetic mutation?
In many genetic diseases, a gene has a mutation, or change, in its instruction manual. That leads to a problem making one of these very important proteins.
How does gene therapy address a mutation?
In gene therapy, a new genetic material is delivered to the cell. That new gene, which is usually engineered in a lab, is different from the person’s naturally occurring DNA.
This new gene is called a transgene and its role is to provide a set of instructions to tell the cell to make a new protein. This new protein is intended to help slow or stop disease progression.
So, it’s not intended to change a person’s genes?
No. The transgene is designed to sit inside a cell’s nucleus but not become a part of the DNA.
Let’s review!
- A gene is a short DNA segment.
- It tells cells how to make a protein.
- In many genetic diseases, the gene is faulty.
- Gene therapy delivers a working gene.
- It sits in the cell nucleus but is not designed to change the DNA.
If you have questions about gene therapy, talk to your healthcare provider or email our Patient Affairs team at [email protected].