Thanks to the ongoing breakthroughs in genetics, a new method of genetic therapy promises to revolutionize the way many fatal diseases and health conditions are treated. Currently, this is one of the most active fields of medical research, and scientists all over the world are beginning to empirically document the therapeutic prowess of this form of gene expression regulation in humans and animals. Gene silencing is a technique whereby specific genes are 'silenced'. In other words, the genetic expression of the targeted genes is reduced - typically, by at least 70%. This allows scientists to not only study how certain diseases are intertwined with gene expression but also develop therapeutic solutions for these conditions. Gene silencing offers tremendous benefits for medicine and the health of human populations. Here are a few examples:
Gene silencing drugs: This is a very straightforward application of gene silencing. A recent demonstration of its therapeutic prowess on humans can be seen in the cholesterol-lowering effect of a new drug called Inclisiran. Being the fruit of the labor of researchers at the Imperial London College, this is one of the first drugs based on the RNA interference therapy (RNAi). On a phase II trial on humans, Inclisiran injections successfully halted the expression of genes responsible for high cholesterol, leading to a LDL cholesterol reduction of more than 50% in most patients. According to scientists, Inclisiran is a much better alternative to statins given that it has virtually no side effects and is perfectly safe to patients (both can be used together). It offers an effective and safe way to lower the risk of cholesterol associated cardiovascular diseases like strokes and heart attacks.
Similar drug-based solutions could help treat or even cure other health conditions. A recent study done on mice - financed by the National Institute of Neurological Disorders and Stroke - has shown that a gene-silencing drug injection can help treat neurological diseases such as spinocerebellar ataxia type 2 and amyotrophic lateral sclerosis (both associated with mutations in the ATXN2 gene). Here, scientists modeled the drug (oligonucleotides) based on antisense therapy principles.
Non-GMO and pesticide-free crops: The health benefits of gene silencing are not limited to the medical field. Given that much of the plant food humans consume is crop-grown, the economic, environmental and health advantages of a crop that is impervious to pests without relying on chemical pesticides or genetic modification are significant. This is what a team of scientists from the Universities of Surrey and Queensland have made possible with the development of 'BioClay'. This spray can be used to silence the expression of certain genes in plants, making them less vulnerable to pests and pathogens that reduce up to 40% of crop output worldwide every year. BioClay is made of clay nanoparticles with RNA molecules. A single application of this non-toxic and degradable solution protects plants for a minimum of 20 days.