Gene therapy uses genes to treat or prevent disease. In the future, gene silencing can be used to treat common health problems.
Recently, UK’s NHS has approved a new cholesterol-lowering jab based on gene silencing technique. This jab will be administered to 300,000 people over the next three years, marking the first use of gene-silencing to treat common diseases.
The cholesterol-reducing drug, called Inclisiran will be injected into patients twice a year. The target patients are those suffering from genetic high-cholesterol conditions, who have suffered a heart attack, or the ones who do not respond well to cholesterol-reducing drugs.
Drug targets a certain gene to prevent it from making the protein that it produces
The gene silencing technique works by stopping a specific gene from producing the protein responsible for a certain illness altogether.
Inclisiran targets mRNA to reduce cholesterol levels in the body by stopping the production of a protein known as PCSK9. This protein normally regulates the cholesterol levels but is found in surplus in people experiencing high levels of LDL cholesterol. So by stopping the production of this protein, the cholesterol levels would dip naturally.
