There is more hope now than ever before that researchers will be able to develop a treatment for FTD. Much research is underway to understand FTD better, treat the symptoms, or change the course of the disease. AFTD and the FTD Disorders Registry want to help you better understand the types of potential therapies that are being tested, such as gene therapy, a promising therapeutic approach receiving increasing recognition from researchers.

Recent advances in our understanding of the genetics of FTD have led to several emerging technologies to treat FTD caused by specific genes. Gene therapy is a promising tool being leveraged to potentially slow or delay progression, with the goal that someday, gene therapy may prevent symptoms from developing. To help you and your family understand gene therapy, AFTD has created a resource to explain the methods and goals of gene therapy.

Gene therapy is not just one technique; there are several approaches to gene therapy. The goal is often to deliver genetic material to compensate for the person’s cause of FTD. For example, if the cause of a person’s FTD is that there is not enough of a certain protein, gene therapy tries to increase its production. If a dysfunctional protein is being produced, then gene therapy may try to silence it.

In addition, other techniques are being studied to use the power of genetics to treat FTD, offering promise for even more options in the future. What scientists are learning from research on genetic FTD, such as how to combat pathological protein accumulation, is helping them better understand and plan interventions for sporadic FTD as well. Please review AFTD’s website to learn more about FTD, the genetics of FTD disorders, and opportunities to participate in research.