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CALL FOR PAPERS     SUBMISSION LAST DATE      28th October, 2020 FOR NOVEMBER ISSUE     

July,2019 Issue

1.GENE THERAPY FOR CARDIOVASCULAR DISEASES

Anurajini Rathnamali*, Luxmiga Tharmarajah

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SRI LANKA

Abstract:

Gene therapy is the insertion of genes into an individual’s cells and tissues to treat disease using experimental techniques. This was done by replacing a mutated gene that causes disease with a healthy copy of the gene and when in activating a mutated gene that is functioning improperly(Müller, Katus and Bekeredjian, 2017). Gene therapy in cardiovascular disease must be aimed at correcting key molecular mechanism in cardiac tissue. This requires introduction of DNA/RNA that targets specific cardiomyocyte processes that alter the cardiovascular disease outcomes(Lowenstein et al., 2000).
One of the key proteins defective in heart is SERCA2a. SERCA2a expression and functions are decreased in cardiovascular diseases. This decease reduces calcium transient that is characteristic of systolic heart disease. The Calcium Upregulation by Percutaneous administration of gene therapy in Cardiac Disease (CUPID) trial looked at the safety and efficacy of SERCA2a in cardiac gene therapy(Murphy et al., 2010). 
In cardiovascular gene therapy, viral vectors genetically modified retroviruses, lentiviruses, adenoviruses, adeno-associated viruses are used. Viral vectors are made replication deficient to ensure safety, but require large amounts of vector particle for efficacy (Williams et al., 2010).