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Heart disease continues to be one of the biggest health threats around the world. It is caused by high cholesterol, which in most cases will result in blocked arteries, heart attacks, and strokes. A new treatment that could alter the way the level of cholesterol is regulated is now underway, as scientists are developing a one-time gene therapy that may result in lifelong control of bad cholesterol levels.
According to a recent study published in The New England Journal of Medicine, scientists have been striving to develop a gene therapy which will address the actual cause of high cholesterol. This new approach does not require daily pills or a diet; instead, it entails only one treatment that cuts or silences some genes in the liver that would otherwise form bad LDL cholesterol.
Recent experiments on gene-editing systems such as CRISPR and the RNA-based ones have provided the most promising results. The mechanisms behind these treatments are to inhibit the operation of the PCSK9 gene which usually raises the levels of LDL cholesterol in the body. The level of cholesterol can be reduced, in response to one treatment, by almost 50 per cent or more by the silencing of this gene.
The treatment is that of imparting genetic instructions into the liver cells either by injection or an innocuous virus. When it enters the body, it alters the mechanisms in the liver for producing cholesterol. Created in a more basic expression, it literally reprograms the liver to maintain the cholesterol level at a healthy level without necessarily taking long-term medication. This may be a life changer for those individuals experiencing high cholesterol levels as a result of genetic factors or being unable to use statins, which are the most prescribed cholesterol drugs.
Human trials have been safe and effective so far. In another study, the subjects under the treatment had their LDL cholesterol level significantly reduced in weeks, and the effects were long-term (months or even longer).
Experts are of the view that the therapy may be given the green light in the coming years given increasing testing and safety check-ups. It is also a hope of the researcher that it would not only assist the high-risk people with heart disease but also stop them before they begin.
If proven successful, this treatment will be a one-time intervention that might help to eliminate the necessity of a lifetime medication. Millions of individuals today reside on statins or other cholesterol medications that they are obliged to take each day and may have side effects. It would provide better control and reduce the number of hospital visits, since a permanent or long-term solution would be involved.
Scientists, however, warn that it is essential to have more data in the long term to ascertain safety, cost and accessibility. Although the therapy is very costly nowadays, with technological advancement it can be cheaper.