Currently, people with high cholesterol face complicated treatment plans, usually taking a range of medications for the rest of their life. This could soon be a thing of the past as a one-time injection could potentially silence the gene responsible for cholesterol, PCSK9.
A new study found this worked just as well as current cholesterol treatments despite having just one dose instead of a constant supply. While gene editing has been considered as a possible treatment for high cholesterol before, there were safety concerns as the methods available could permanently alter other parts of the DNA.
This can cause a wide range of issues from genetic disease like Down Syndrome to autoimmune problems. However, using epigenetic editing methods instead researchers could modify the gene without affecting the DNA.
This new therapy is delivered via a single intravenous infusion. The method adds certain molecules into the body that effectively turn off the PCSK9 gene only, according to News Medical Today.
Researchers behind the study published in the Nature Medicine journal have undertaken a number of trials with human liver cells, mice with human PCSK9 genes and non-human primates. This final trial showed great success, cutting cholesterol levels by around 70% as PCSK9 levels dropped by nearly 90%.
The mice studies proved the treatment was sturdy and long-lasting too. A single treatment cut PCSK9 levels by 98% and showed a “substantial decrease” in bad cholesterol over a year.
Astonishingly, researchers were also able to reverse the effects of the treatment in mice, returning their PCSK9 gene to normal. This adds an extra safety measure proving that the treatment can be undone if necessary.
In the non-human primates, they did notice some minor impact on other parts of the DNA. But this didn’t change how the affected genes worked so it helped quell concerns.
This therapy could be particularly useful for people who have familial high cholesterol. Family history may mean they have extremely high cholesterol levels from a young age as their body can’t properly regulate their cholesterol.
Unfortunately, people with high cholesterol may not be seeing this treatment any time soon as researchers emphasised that clinical human trials and “extensive safety testing” is still needed. If it is proven safe this could be revolutionary for high cholesterol patients, greatly minimising the burden their health condition has on their daily life.