After Jeffrey was diagnosed, we travelled all over the world to get answers—Jeffrey could have anything he wanted, but we couldn't give him his health. He underwent some of the most innovative treatments from the top doctors in the world, but science hadn’t caught up with us in time.
Now, there are several different types of treatment available for patients with even the most severe PIs. As part of a new series on our blog, we’ll be providing an overview of different treatments so that you, our community, can learn more, stay informed, and have access to the best information at your fingertips.
Today, we want to explain Gene Therapy. Gene therapy is a type of medical treatment that uses a person’s own genes as a method to treat their disease. Researchers and investigators approach this type of therapy in a variety of different ways, such as replacing a mutated gene with a new gene, inactivating a mutated gene that is not functioning properly, and introducing a new gene in order to fight disease.
Gene therapy can be used to treat a growing list of PIs, and based on your physician’s recommendation, may be the appropriate treatment protocol for specific disorders. We have witnessed an increased success rate of this therapeutic benefit for a range of PIs, and look forward to tracking future research progress in this area.
True to our mission, early diagnosis remains the best way to prevent severe infections and improve a patient’s outcomes and quality of life. We are grateful for the scientific progress surrounding treatments over the past thirty-five years, and hope that with continued progress, Jeffrey’s story will be rare among PI patients.