Tuesday, June 24, 2014

Heart Attack Vaccination Could Be on the Way

This post was contributed by @MikeMadarasz

Heart attacks are the leading natural killer worldwide and claim approximately one million lives every year.  A staggering figure, researchers with the Harvard Stem Cell Institute and University of Pennsylvania claim to have discovered a treatment that could potentially reduce the risk of heart attack by up to 90%. 

Scientists at the Harvard Stem Cell Institute and University of Pennsylvania have collaborated on a “genome editing” approach that has dramatically reduced cholesterol levels in mice with a single injection.  Kiran Musunuru MD, PhD, leader of the HSCI team working on this project, called the success of the first iteration of the experiment “pretty remarkable”.

The research was focused on altering a gene known as PCSK9 which is found in the liver and recognized as being a regulator of cholesterol.  In 2003, a group of researchers in France discovered that a rare mutation in this gene was positively correlated with high cholesterol and early heart attacks. 

More recently, a research group in Texas has discovered that a separate mutation in this gene also has the opposite effect.  The “good” mutation is present in only 3% of the population, but those carrying this specific gene have bad cholesterol levels ranging from 15-28% lower.  Consequently, their risk of heart attack is 47-88% lower. 

This lead Musunuru and his team to deduce that if they could replicate the effect, they’d be able to protect people from heart attack.   

The research reached a turning point in 2013 with an improvement to a technology called Cas9.  Said Musunuru, “Cas9 is a protein that will create a break in DNA, and the CRISPR is an RNA component that will bind to a matching sequence and directs the Cas9 to that sequence in the DNA in which you are interested. This creates a break where you want it. The cell can then repair itself, though often with errors, which is useful if you want to disrupt a gene to create a ‘knockout’ of the gene.”

“What we were thinking was that with this genome editing technology we can do something we couldn’t do before—make permanent changes in the genome at the level of the DNA; we can actually go to the source. So the question was whether we can use genome editing to make normal people like people born with the ‘good’ mutations.” Based on the results with mice, the answer is a resounding “yes”.

The group saw a 35-40% reduction in cholesterol levels in these mice which they say could translate into about reduction in heart attacks as high as 90% in humans.  Says Musunuru, “It could be a one-time treatment, a permanent alteration. If you used this in a population, you could reduce the occurrence of heart attack by 30 percent, 50 percent, 90 percent.”

Get the full article from HSCI here.  

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