GENE THERAPY FOR CARDIOVASCULAR DISEASES

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).