Gene therapy canoffer best results in Danon disease treatment, according to new research. Inthis rare health condition, human body is not able to perform the basicbiological work of removal and reuse of proteins. Thus, this situation badly affectsindividuals various body parts. It includes liver, skeletal muscles, eyes, neurologicsystem, and heart. In addition, many patients die or have to go through hearttransplants in their 30s.

Finding Therapies That Help in Addressing Root Cause of Disease

Scientists fromthe University Of California San Diego School Of Medicine revealed the outcomesof their latest research. They stated that they have discovered an advancedtechnique for the treatment of Danondisease.Basically, thistechnique uses gene therapy. The latest study is open for access inthejournal Science Translational Medicine.

Eric Adler isthe key investigator of the research. He stated, Heart transplant cannotbe 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 therapiesparticularly aimed at addressing the root cause of this disease. In the latestresearch, we utilized mice that were used as a model for Danon disease. Thesemice were missing this particular LAMP gene. We applied gene therapy to this micegroup. Further, the results were compared to mice that did not get treatment.

In the outcomesof this research, mice receiving gene therapy offered positive results. Thefunctioning of liver, heart, and muscle in these mice was improved. Inaddition, there was improvement in the hearts overall function of ejectingblood and relaxing. There was improvement in the bodys ability of proteins degradationand metabolism.

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Tapping Gene Therapy Technique in Danon Disease Treatment - TMR Research Blog

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