The genes inside your body contain the DNA that controls much of who you are from your hair and eye color to the function of your body’s systems. However, sometimes these genes malfunction or develop incorrectly from birth causing disease. Researchers are now studying new treatment options for these kinds of diseases that involve repairing, replacing or altering genes in order to cure or improve the body’s ability to fight disease.
What is Gene Therapy?
Gene therapy is an emerging treatment option that utilizes the body’s genes to cure or fight disease. Treatments may involve repairing or replacing the body’s own genes or using genes derived from an external source. The new therapeutic genes are introduced to the body during gene therapy through a vector (carrier) which is made from a reprogrammed virus, stem cells or fatty particles called liposomes. Each type of gene therapy is performed differently so the details of the procedure will vary depending on the disease type and therapy used.
There are three main types of gene therapy:
- Inactivating the mutated gene that is causing disease
- Replacing the mutated gene that is causing disease with a healthy copy
- Introducing new or altered genes that can help train the body’s immune system to fight a specific disease
What is Gene Therapy Used to Treat?
Researchers have only begun to scratch the surface of what is possible for gene therapy as a treatment option. Currently, gene therapy is an FDA-approved treatment for:
Researchers continue to be hopeful that gene therapy may one day be used to treat a wider range of diseases including heart disease, cancer, diabetes and more.
Gene Therapy Treatments
There are currently five FDA-approved gene therapy treatments available in the United States. Three are approved for adults while two are approved for pediatrics only. Most gene therapies have very specific guidelines that determine eligibility. Speak with your physician to determine if you are a candidate for gene therapy treatment.
The treatments currently available are:
- Hemgenix® – for adults with hemophilia B
- Luxturna® – for adults with inherited retinal disease
- Skysona® – for pediatric patients with active cerebral adrenoleukodystrophy (CALD)
- Zolgensma® – for pediatric patients with spinal muscular atrophy (SMA)
- Zynteglo® – for adults with beta-thalassemia, an inherited blood disorder
Why Choose Penn Medicine for Gene Therapy?
At Penn Medicine, our researchers and medical professionals strive to continuously be at the forefront of medical innovation. As an academic medical institution, our providers often have access to the latest techniques, therapies and clinical trials, enabling them to extend this access to their patients.
Gene Therapy Clinical Trials
Though the FDA has approved only a few gene therapy treatments, many more studies are ongoing. Gene therapy is currently being studied to treat many diseases like cancer, heart disease, cystic fibrosis, sickle cell disease and hemophilia A.
At Penn Medicine, you have access to leading clinical trials studying new and innovative treatments and therapies for a wide range of diseases. Participating in clinical trials gives patients access to new treatments that could potentially help improve their condition while being involved in critical research that could advance the treatment of their disease in the future. Speak to your provider to determine if you qualify to take part in a clinical trial.