Gene therapy might lead to a one-time vaccine-like treatment that would permanently lower bad cholesterol and reduce the risk of heart attack by as much as 88 percent.
Heart disease is currently responsible for about one in every four deaths in the United States, adding up to more than 600,000 deaths each year.
According to the Harvard Stem Cell Institute, however, new genome research shows great promise of permanently reducing the risk of heart attack.
Work on this project first started in France in 2003 when researchers discovered that a specific gene in the liver, PCSK9, seemed to be responsible for cholesterol regulation. Some families with a mutation of this gene had very high cholesterol and were very likely to have early heart attacks.
Meanwhile, in Texas, another research group identified a population that had a different kind of mutation of PCSK9 — this time a very good mutation. This mutation produced the opposite effect. People with the mutation have very low levels of low-density lipoprotein (LDL or bad) cholesterol levels. What’s more, they were significantly less likely to have a heart attack.
A recently developed gene editing technology called CRISPR/Cas9 allowed these researchers to alter the PCSK9 gene in mice to convert it to the good version that would potentially help them live longer. This change caused the liver to stop producing a particular protein that would prevent the removal of cholesterol in the bloodstream. It also solved the problem that traditional cholesterol drugs have – they don’t last very long. Rather than constantly having to get shots or take medicine to reduce cholesterol, scientists hypothesize that it may be possible to have a lifetime change in effect with just one application.