I’ve heard that gene therapy is soon going to treat all kinds of medical conditions. What is it?

While gene therapy sounds like something straight out of a science-fiction movie, it’s actually the next game changer in medical treatment. Gene therapy involves altering certain genes inside your body’s cells in an effort to cure a disease or stop it from progressing.

Genes are made up of DNA, or deoxyribonucleic acid, which acts as blueprints for building the enzymes and proteins that make our body work. We typically get two copies of each gene from our parents, which influence everything from the colour of our hair to how well our immune system works. Through gene therapy, a small adjustment is made that changes a person’s genetic makeup with the goal of treating or curing a disease. This can be achieved by:

• Replacing genes that are causing disease
• Disabling genes that aren’t functioning properly
• Introducing new genes

Gene therapy holds promise for treating, and perhaps eventually curing, a wide range of diseases, but it’s still early days as more research is needed on its long-term effects.

There are currently two gene therapies available to treat cancer approved in Canada, and it’s anticipated that the majority of gene therapies will focus on cancer treatments and rare inherited genetic disorders, such as blood disorders, neurological conditions affecting the brain and spinal cord, eye diseases, and muscle disorders. However, in future, even common conditions like heart disease and diabetes may be treated through gene therapy.