Medizinische Universität Graz Austria/Österreich - Forschungsportal - Medical University of Graz
Gewählte Publikation:
Zotter, K.
Gene therapy in sudden cardiac death: Current status and future therapeutic perspectives
Humanmedizin; [ Diplomarbeit ] Graz Medical University; 2019. pp. 73
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- Autor*innen der Med Uni Graz:
- Betreuer*innen:
- Rupp Verena
- Windpassinger Christian
- Altmetrics:
- Abstract:
- Introduction: Sudden cardiac death is defined as a sudden, unexpected death due to cardiac causes and represents the most common fatal manifestation of heart disease. In young patients under 35 years, a common cause of this is congenital heart disease. The current treatment options, such as organ transplantation or implantable defibrillator, are due to some side effects or contraindications insufficient treatment options. Future therapy concepts of sudden cardiac death are currently the subject of research and show promising approaches. Methods: To investigate this topic, a comprehensive literature research was conducted with comparison of the statements from different sources and critical analysis. Results: One way to curatively treat terminal heart failure is the bioengineering of human myocardium on native extracellular matrix as an alternative to donor organs. The goal is to create a whole bioengineered heart that is built by using a decellularized scaffold which then gets recellularized by billions of multiple types of cardiac cells. Furthermore, treating diseases by fixing the mutant gene or normalizing the expression of overactive/underactive genes by means of genome editing is a future treatment option. Particular attention is placed on the introduction of specific genetic mutations into human pluripotent stem cells by means of CRISPR/Cas9 technology and delivery of these via adeno-associated-viruses into the target cells. In addition, cardiac regeneration by proliferation of differentiated cardiomyocytes and cardiac optogenetics are promising therapeutic methods. Conclusion: The application of these forms of therapy has not yet found its way into the clinic, but thanks to intensive research, it holds promising prospect.