What is gene therapy?
Gene therapy is a medical approach that uses genetic material to prevent and treat disease. The technique allows healthcare providers to treat certain conditions by changing your genetic makeup instead of using traditional treatment methods like medication and surgery. In this way, providers can address the underlying cause of the disease or instruct your own body to mass-produce desirable medication or proteins.
In gene therapy, genetic material is transferred to your cells. This genetic material then changes how your cells produce proteins. It can:
- Reduce levels of certain disease-causing proteins.
- Increase production of working proteins.
- Produce new or modified proteins within a cell.
How can gene therapy help me?
Your body’s genetic information is kept in chromosomes inside the nucleus of your cells. Each chromosome is made up of DNA that stores information to determine your unique traits. Specific sections of DNA are called genes. Genes provide instructions for how to make proteins. Proteins play an important role in how your body functions. A small change to the DNA within your genes can alter how proteins work.
Gene variants (genetic changes) occur as cells age or after exposure to certain chemical or environmental factors. Cells often recognize these genetic changes and repair them. Other times, they can cause a disease or disorder and you‘ll need treatment. Your biological parents can also pass along these gene variants, causing disease from an early age. By using gene therapy, healthcare providers aim to address the underlying cause of disease. If genes are like the blueprint of your body, gene therapy can fill in missing parts or correct errors in the drawings.
What is gene therapy used for?
Most gene therapies are still in the clinical trial phase. Clinical trials play an important role in finding treatments that are safe and effective. Clinical trials are investigating gene therapy for the treatment of cancer, macular degeneration and other eye diseases, certain genetic conditions and HIV/AIDS.
The U.S. Food and Drug Administration (FDA) has approved two gene therapies for use in the U.S.:
- Luxturna®: Approved in December 2017, Luxturna is a one-time treatment used to improve vision in people with genetic vision loss due to certain inherited retinal (eye) diseases.
- Zolgensma®: The FDA approved Zolgensma in May 2019 to treat spinal muscular atrophy in children younger than 2 years old.
