"With this genome-editing technology, we can make permanent changes in the genome at the level of the DNA, he added."
Washington, June 11 - Can you imagine a single shot that can lower cholesterol levels to an extent that the risk of heart attack can be reduced by 40-90 percent?

Scientists at Harvard Stem Cell Institute (HSCI) along with researchers at the University of Pennsylvania have developed a genome-editing approach for permanently reducing cholesterol levels in mice through a single injection.

You have people who have won the genetic lottery. They are protected from heart attack, and there are no known adverse consequences. So that led us to reason that if we could find a way to replicate this, we could significantly protect people from heart attack, explained Kiran Musunuru from HSCI.

PCSK9 is a gene that works as cholesterol regulator.

But some people have mutations in PCSK9 that have the opposite effect.

Those with the mutations have low-density lipoprotein (LDL or bad) cholesterol levels about 15-28 percent lower than the average level.

And the people with that good defect have heart attack risks that range from about 47-88 percent below average.

Musunuru and his team project to turn normal PCSK9 genes into those with the good defect.

The PCSK9 gene is expressed primarily in the liver.

It produces a protein that is active in the bloodstream and prevents the removal of cholesterol from the blood.

The main option for reducing cholesterol is statin drugs such as Lipitor but many people taking statin drugs every day still have heart attacks. So there is still a great need for other approaches, Musunuru noted.

With this genome-editing technology, we can make permanent changes in the genome at the level of the DNA, he added.

The research was published in the journal Circulation Research.


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