What is gene therapy?
Gene therapy is a technique used in an effort to treat or prevent disease. When a gene mutation (a permanent alteration in the DNA sequence) causes a protein to be missing or faulty, gene therapy may be able to restore the normal function of that protein. There are several different approaches to gene therapy:
- introducing a new or modified copy of a gene
- turning on or off genes to avoid disease
- replacing the disease-causing sequence of a gene with a healthy copy of that sequence
The goal of gene therapy is to change the course of disease by targeting its genetic cause.
Understanding genes
Genes come from our parents. They dictate everything from the color of our eyes to our immune system to our likelihood to develop diseases. Genes contain the information needed to build and maintain cells by encoding proteins.
A gene is a small section of a cell’s DNA, consisting of four chemicals called bases. These bases form the backbone of DNA and — much like a manual — provide instructions for assembling proteins, the building blocks of the human body, such as the muscles, organs and immune system.
The trouble is genes aren’t always built correctly. When this happens, it changes the gene's DNA. This is called a mutation. Mutations can be passed down from someone's parents or can occur for the first time in that person. A mutation in the gene can alter how proteins work. Sometimes, mutations can cause disease. That’s where gene therapy may help.