Key result
Genetic therapies, including gene therapy and antisense oligonucleotides, have been successfully implemented to lower triglyceride levels in patients with hypertriglyceridemia.
This review highlights the rapid progress in using genetic information, gene therapy, and antisense oligonucleotides to treat hypertriglyceridemia and understand cardiovascular risk.
Hypothesis-generating for genetic TG-lowering therapies; prospective RCTs needed before clinical adoption.
The use of genetic information to explore and treat diseases is ever–expanding, varying from the use of classical approaches for monogenetic disorders to the growing genome–wide association studies to understand more complex traits. In hypertriglyceridemia, development has progressed rapidly. We have now observed the use of genetic informationtotreatmonogeneticdisordersusinggenetherapy,which,forthefirsttime,hasbeenimplemented successfully in human subjects. In addition, antisense oligonucleotide therapy in mice and more recently, in humans, has been demonstrated to lower triglyceride levels. In polygenetic disease, the use of large–scale genome–wide association studies has changed our perception of the underlying phenotypes, demonstrating a large overlap in common genetic determinants. This information is translated to understanding reactions to drug therapy, but also in relation to environment interaction. Finally, the use of genetics for predicting cardiovascular disease risk is being continuously studied, although clinical application appears to still be far along the road ahead.
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Moens et al. (2013) conducted a review in Hypertriglyceridemia. Genetic therapies (gene therapy and antisense oligonucleotides) was evaluated. Genetic therapies, including gene therapy and antisense oligonucleotides, have been successfully implemented to lower triglyceride levels in patients with hypertriglyceridemia.
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