In the first of its kind, experimental gene editing has paved the way to eliminate heart diseases. These genes are responsible for heart diseases, and eliminating them will reduce the risk of heart diseases. Moreover, these genes are also carried by human beings as well, which raises bars of hope to eliminate the disease in the future. However, we do not know how this might work as the study is in its initial stages, and we need to study a lot about its effects on monkeys. Given the history of gene-based treatments having mixed criticism, it will definitely take longer to get into our commercial market.
The results were published in a virtual conference for stem cell research. The goal was set out to block two genes that regulate cholesterol metabolism on the liver. People who inherited naturally mutating versions that destroy these genes suffered less from heart disease. People with higher levels of LDL and triglycerides suffer from all heart diseases to stroke. Due to this importance, drug companies are already marketing gene inhibitors that lower LDL and triglyceride levels. But these inhibitors are expensive, and therefore scientists wanted to make it cost-effective and affordable.
This is where Dr. Sekar Kathireasan at Verve Therapeutics comes into the picture. He decided to edit the genes instead of injecting inhibitors regularly. They created an RNA editor that will effectively bind to cells and change the genetic sequence. This editor is injected in the form of tiny spheres to protect from degradation. Once they reach the target site of the liver, they bind to cells and change the genetic sequence. Once edited, they remarkably bring down the lipid levels as max as 59 percent. This worked on over 23 monkey models, which are very tough to get such results when compared to mice models. In addition, they also brought down the triglyceride levels up to 64 percent.
Although the editor is said to work on monkeys, we do not know the side effects of the editor till now. Sometimes these gene modifications can bring about deleterious effects in the host. Therefore, experts are slightly concern about this aspect. Fortunately, these monkeys do not show any adverse effects until now. In addition to this, the scientist also needs to evaluate the lasting effect of this editor. Experts hope this can be one time change and need not be done again.
But we need clinical trials and research to prove it. If everything goes, Dr. Sekar hopes this technology might be available for humans to fight heart diseases. Also, this will help third world countries where people cannot afford expensive treatments on a regular basis. With higher hopes, this can this therapy can be groundbreaking to combat to treat deadly metabolic diseases? Further research will give us more answers.
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Source: NY Times