New studies reveal that one in four deaths in the United States is the result of heart attack. The numbers are high with an annual rate of over 700,000 victims of heart attack in the nation annually. The Centers for Disease Control and Prevention (CDC), rank this as the leading cause of death within our borders. A new technology known as gene editing may offer a change in the susceptibility to heart disease for some people.
Lifetime risk for heart disease
There are multiple factors that influence the risk for developing heart disease for an individual. The majority are due to lifestyle choices, along with genetic influences inherited from parents. Lack of exercise, improper diet along with smoking increase the odds of developing heart disease. Abandoning unhealthy behaviors and adopting a fitness centered lifestyle can lessen the chances of heart disease, but the practice of gene editing shows promise in further reducing risk.
The culprit signaling risk
A specific gene that is called ANGPTL3, assists in the regulation of triglycerdes, or fats in the blood. An abundance of triglycerides ups the odds of heart disease development. Natural mutations occur in the gene when influenced by unhealthy lifestyle factors that increase the production of triglycerides. Diet changes along with prescribed medications can cut the risk, but the ANGPTL3 mutation is shown to have a greater positive effect for lowering risk.
Is gene editing safe?
According to the available data, gene editing does not present any known side effects. Testing has been ongoing with researchers based at the University of Pennsylvania taking the lead. They’re reviewing thee results of the recent gene editing technique that offers new hope for those predisposed for cardiovascular disease.
The first phase of the journey
Initial data collected from research into gene editing has been published in the “Circulation” journal. Base editing is the term for the CRISPR inspired technique. The procedure involved a study of healthy mice injected with the base editing treatment which modifies the ANGPTL3 gene. Comparisons of the fat levels in the blood of the treated animals were made with mice who had not received the gene editing treatment. The results showed a lowering of the blood fat of thirty percent in the treated animals.
Determination of efficacy in humans with inherited disorders
A rare genetic condition known as homozygous familial hyperchcolesterolemia places those inheriting the disorder at a severely high risk for heart disease with few effective treatment options. Physicians speculate that turning off the gene responsible may be a life saving treatment that forcibly brings down triglyceride levels.
The CRISPR experiment
The CRISPR technique was employed to test the hypothesis that gene editing may offer hope for those who inherit genetics that predispose them to heart disease as discussed above. A mouse model was created specifically for the homozygous familial hypercholesterolemia disorder. Base editing treatment was applied to the mice in the study, and two weeks later, the mice were tested to observe the results. Testing showed that triglycerides were reduced in some cases by up to fifty-six percent when compared to the mice that were not treated with base editing. This showed initial evidence that modifying the gene is effective in treating this particular rare genetic condition.
The current status of the research
Research into gene editing, so far, has been strictly limited to testing in mice. It is unknown what the results will be for humans, but it offers an indication that there is potential for a step forward in identifying a treatment for rare genetic diseases. Not all humans respond to lifestyle changes or medications, leaving them with no treatment options for high triglycerides. A breakthrough in alternative treatment options could save the lives of many who currently live with severe risk.
The next step
The research team is currently making preparation for work that will bring them closer to trials with humans. The precursory testing will soon begin. Researchers plan to inject mice with genetic materials from human liver. This will be used to assess effectiveness and safety potential on ANGPTL3 genes in humans.
The future of genetic engineering in humans
If success is achieved as expected, the next step will be clinical trials with people. The possibility of developing a CRISPR vaccination that need only be given once, could be as close as just five years in the future. There is hope that one day, genetic predisposition to disease could be altered through gene editing therapies and we’re currently learning how human gene engineering may offer hope to those who currently have few options.