Gene Therapy Experts Plan to Slash Heart-attack Risk With Single Injection

Updated July 11, 2019

We live in an age where medical breakthroughs are the new normal. But there are some serious illnesses, like heart disease, that defy the steady advance of science. Still, scientists may recently have found a way to lower the risk of heart attacks with a single injection. 

Medical researchers now aim to set up trials for gene therapy within the next three years. This new approach may be the final solution to treating heart failure. 

Heart Diseases Impact Hundreds of Thousands in the U.S. Every Year 

Human beings have suffered many distressing diseases throughout history, but in modern times, heart attacks and strokes head the list of the most fatal diseases.

According to the U.S. health protection agency, the Centers for Disease Control and Prevention, 790,000 Americans get a heart attack every year. It’s the first time for about 580,000 of them. The remaining 210,000 have had attacks before. 

Those at risk are put on medications they have to take for the rest of their lives. Doctors and patients have resigned themselves to the inevitable fact that heart conditions require chronic care.

Patients may have to take tablets for high blood pressure or blood thinners or be put on statins to lower cholesterol. 

Genetic Therapy to Reduce Heart Disease Risk 

Yet all this may be about to change in an unexpected way. Medical researchers in the United States have plans to use genetic therapy to reduce the risk of heart attacks.

More dramatic still, they hope to achieve this lofty goal with a single injection that will precipitate a biological chain reaction of genetic transformation, a process called gene-editing. 

It may take three years to complete the clinical trials. Researchers will focus on people with a rare genetic disorder that makes them susceptible to heart attacks at a relatively young age, during their thirties and forties.

Once researchers can prove that the treatments are both safe and effective, they plan to extend their studies to include a wider range of people at risk for heart attacks or strokes. 

Reduced Health Care Costs 

Besides transforming the lives of those with heart disease, this radical new procedure may also transform the health care system itself by drastically reducing the cost of treating these conditions.

It will reduce the cost of heart health care by replacing the chronic care model with a single effective treatment. After receiving the injection that precipitates gene editing, a patient theoretically will no longer have any heart problems. 

In an article in the U.S. Edition of the Guardian that discussed how gene editing could reduce the risk of a heart attack, Harvard Medical School cardiologist, Sekar Kathiresan, who is leading the experimental trials, playfully refers to his one-time treatment plan as a “one and done” approach. 

What Is Gene Editing?

The experiment begins with a patient receiving an injection of nanolipids in their arm. Nanolipids are nano-sized fat particles. (A “nano” is short for “nanometer.” This is one-billionth the size of a meter.

In comparison to a nano particle, the size of a human DNA strand is more than twice as large. Human DNA is 2.5 nanometers wide.) 

Because of their microscopic size, nanometric fatty spheres will target liver cells, penetrate them, and release Crispr-Cas9.

Crispr-Cas9 is an RNA-based molecule with Cas9, a bacterial enzyme, and its mission is to target a specific area on a DNA double helix. Scientists use it as a gene-editing tool. 

Once Crispr-Cas9 locates a designated DNA strand, it goes to work to cut the DNA at an exact place — like a pair of scissors cutting a ribbon at a marked point. Using this gene-editing tool, scientists can rearrange genetic code, cutting, pasting, and deleting as required.

Once released from a liver cell, Crispr-Cas9 goes in search of PCSK9, a gene which triggers heart attacks; and once it finds it, Crispr-Cas9 switches it off.

Relevance of Radical Gene Therapy 

Although it may seem that gene editing therapy will only work for individuals who have a genetic predisposition for heart failure, those involved in developing this therapy believe that, in time, they will be able to work out how to apply this therapy to anybody at risk for heart disease.

So, after the research expands after the success of initial trials, it could be possible to help many types of patients at risk for cardiac arrest.

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