Gene Therapy

Gene therapy is a treatment for genetic disorders caused by gene mutations (when a gene does not work correctly). You can be born with gene mutations or they can arise during the course of your life. At The University of Kansas Center, gene therapy is used in the prevention and treatment of certain types of cancer and other diseases.

What is gene therapy?

We all inherit our genes from our biological (birth) parents. Our genes come with a set of instructions (DNA) that determine physical attributes, such as height and eye color, as well as proteins or the building blocks of organs, immune system and more. You inherit your DNA from your biological parents. If your genes mutate or malfunction, they don’t work properly and can cause a host of diseases including cancer.

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To make an appointment at The University of Kansas Cancer Center, call 913-588-1227.

Who can have gene therapy?

Gene therapies are advanced, first-in-the-world therapies. Currently, more than 40 gene therapies are FDA-approved, 3 of which are used in the treatment of cancers. More gene therapies for the treatment of cancer and cancer prevention will become available to the public as active and future clinical trials continue to progress.

How does gene therapy work?

There are 2 main types of gene therapy: gene addition and gene editing. If a gene does not work properly, is damaged or even missing, gene therapy updates the instructions (DNA) to replace the missing or faulty gene with a healthy copy (gene addition) or mend the faulty gene by editing the genetic material in your DNA to treat the disease (gene editing also known as gene correction).

Other types of gene therapy include:

Gene silencing: stops the development of diseased proteins
Gene reprogramming: Adds new genes to a cell to alter damaged genes
Cell elimination: Destroys cancerous (malignant) cells and prevents excessive growth of noncancerous (benign) cells

Stephon Fisher, a young man with glasses, is pictured smiling while holding a child in one arm with his other arm around the shoulder of his daughter.

Gene therapy gives engineer pain-free life

Undergoing gene therapy reprogrammed Stephon’s ability to produce normal red blood cells.

Read Stephon's story

Benefits and risks of gene therapy

Gene therapy offers a number of clear benefits, including:

Hope where there was none. The strongest benefit outshines all others. Gene therapies have overcome diseases that previously had very limited treatment and recovery options. As advancements continue to refine and develop new and existing gene therapies, treatments will continue to evolve in scope.
One-time treatment. Unlike other therapies that require regular medication, gene therapies often consist of one treatment.
The earlier, the better. Early application of gene therapy can often stop the disease being treated from getting worse.
Treats the disease, not the symptoms. Gene therapy is a targeted treatment that goes straight to the source.

Gene therapy does come with some potential risks. As genes cannot be added directly to your cells, they are delivered using a carrier or vector often in the form of a virus. A virus can detect cells and can insert material into those cells. These viruses are different than the ones we know as they carry genes to stop, rather than cause, disease.

Using a virus to bring gene therapy to your cells can have some risks, such as:

Immune system may attack. Your body may detect the virus and believe it to be a disease-causing virus. In these instances, the reaction can range significantly, from simple swelling to organ failure.
Damage to healthy cells. The virus may enter unintended, healthy cells.
Infection. Once in your body, the virus may cause disease.

Why choose us

The University of Kansas Cancer Center offers comprehensive cancer treatment, including cellular therapies. Our multidisciplinary care teams are dedicated to ensuring you receive the highest quality care and most personalized treatment options available. These care teams work together to develop individual treatment plans for each person based on their needs and goals with input from a variety of specialists.

The University of Kansas Cancer Center is a National Cancer Institute-designated comprehensive cancer center and consistently recognized among the nation’s 50 best cancer centers as ranked by U.S. News & World Report.
Interdisciplinary care means you will benefit from a comprehensive team of doctors from different specialties, all working for you. Our interdisciplinary teams include expert medical oncologists, surgical oncologists, cellular therapeutic specialists, nurses and physical and occupational therapists who work side-by-side to provide you with the best care.
That means we work with you from the moment you walk through our doors, throughout your diagnosis and treatment and beyond.
Because The University of Kansas Cancer Center is part of an academic medical center, we offer innovative treatments and clinical trials – often long before other hospitals can.
Our board-certified, fellowship-trained physicians provide some of the most advanced and efficient treatments to alleviate pain.

We are the only National Cancer Institute-designated comprehensive cancer center in Kansas and 1 of fewer than 60 in the United States. Patients treated at NCI-designated comprehensive cancer centers have a 25% greater chance of survival compared to other cancer centers. NCI-designated cancer centers are pioneers in cancer research, recognized for their scientific leadership, resources and depth of research in basic, clinical and population sciences. To maintain our NCI designation, we go through a rigorous, highly competitive process to demonstrate our contributions to reducing the cancer burden in our region and throughout Kansas and western Missouri.