Gene therapy is an innovative approach to medicine that uses genetic material to prevent, treat and potentially even cure disease. In gene therapy, genetic material is delivered to your cells and changes how your cells produce proteins. While still in its earliest stages, gene therapy has the promise to be a life-changing medical treatment.
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:
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.
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.:
Gene therapy works by replacing or inactivating disease-causing genes. In some cases, gene therapy introduces new genes into your body to treat a specific disease.
With gene therapy, healthcare providers deliver a healthy copy of a gene to cells inside your body. This healthy gene may replace a damaged (mutated) gene, inactivate a mutated gene or introduce an entirely new gene.
The genetic material needs help getting where it needs to go, like being wrapped in a package with an address label. This package is known as a vector. Viruses are usually used as vectors because they’ve evolved to be very good at getting into cells. Scientists use the same ability to deliver the genetic material into your cells. Any disease-causing part of a virus is removed, allowing it to enter your cells without making you sick.
There are two main ways to receive gene therapy: in vivo and ex vivo.
Healthcare providers decide on the right approach based on the best way to target the specific disease.
The use of gene therapy comes with many benefits and risks.
Risks may include:
Researchers are investigating gene therapy to treat many different health conditions, including cancer, eye diseases, some genetic conditions and HIV/AIDS. If you’re interested in participating in a clinical trial involving gene therapy, speak with your provider. They can help determine whether gene therapy might treat your condition.
A note from Cleveland Clinic
There are still many challenges to overcome for those seeking gene therapy as a potential treatment option. One challenge is time — it’s a lengthy process to test potential treatments in clinical trials and then undergo the FDA approval process. But advances in gene therapy research allow us to better understand how we could treat, and hopefully someday prevent, rare and debilitating diseases.
Last reviewed by a Cleveland Clinic medical professional on 12/04/2023.
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