Tapping Gene Therapy Technique in Danon Disease Treatment

Gene therapy can offer best results in Danon disease treatment, according to new research. In this rare health condition, human body is not able to perform the basic biological work of removal and reuse of proteins. Thus, this situation badly affects individual’s various body parts. It includes liver, skeletal muscles, eyes, neurologic system, and heart. In addition, many patients die or have to go through heart transplants in their 30s.

Finding Therapies That Help in Addressing Root Cause of Disease

Scientists from the University Of California San Diego School Of Medicine revealed the outcomes of their latest research. They stated that they have discovered an advanced technique for the treatment of Danon disease. Basically, this technique uses gene therapy. The latest study is open for access in the journal Science Translational Medicine.

Eric Adler is the key investigator of the research. He stated, “Heart transplant cannot be useful while treating the other organs affected in Danon disease. Further, it is not always available for all patients.  This specifies the need to discover new therapies particularly aimed at addressing the root cause of this disease. In the latest research, we utilized mice that were used as a model for Danon disease. These mice were missing this particular LAMP gene. We applied gene therapy to this mice group. Further, the results were compared to mice that did not get treatment.”

In the outcomes of this research, mice receiving gene therapy offered positive results. The functioning of liver, heart, and muscle in these mice was improved. In addition, there was improvement in the heart’s overall function of ejecting blood and relaxing. There was improvement in the body’s ability of proteins degradation and metabolism.