In a new twist of events, aided by small tailor-made pieces of RNA, scientists have been able to successfully lower the levels of “bad” cholesterol in pre-clinical trials by two-thirds using single doses of siRNA.
Researchers have studied people with mutations in the PCSK9 gene “short for proprotein convertase subtilsin/kexin type 9” which is responsible for the production of a protein that raises the level of LDL & have found that they are less prone to hypercholesterolemia & other cardiovascular-associated disorders. In fact, they are 28% less liable to develop coronary heart diseases.
Therefore, eliminating the production of this protein is beneficial for patients suffering from high levels of blood cholesterol. In order to achieve this, little pieces of designer siRNAs were designed which attach upon the cell’s mRNA and put an end to the process of protein translation.
These trials have been performed on mice and rats that have been genetically altered to produce normal human PCSK9 protein end product.
In addition, non-human primates were also included where they showed an average of 56% reduction in cholesterol level, with one of them showing a surprising 70% reduction.
It is worthy to say that drugs available now in the market have only proved successful when taken at maximum doses over prolonged periods of time & showed only 20-50% drop in LDL cholesterol. This opens up a new horizon for patients who have not responded to conventional drug therapy or may be used in combination with the existing medication to produce more promising results.
Tags: cholesterol, PCSK9 gene, siRNA