Scientists have presented the results of a remarkable preclinical study that employs an epigenetic approach instead of traditional gene editing. In this method, DNA sequences are not cut and replaced — instead, scientists merely adjust the activity of specific genes. This treatment has already led to the normalization of cholesterol levels in mice.
Epigenetic editing aims to change the expression of specific genes involved in the development of disease. This approach is more attractive compared to traditional CRISPR gene editing because it reduces the risks of complications due to the undesired consequences of cutting DNA. Epigenetic editing occurs by adding a specific chemical marker — a methyl group — to DNA, to turn a gene off or on depending on the goal.
Using mouse models with high cholesterol levels as examples, scientists demonstrated in Nature that editing the epigenome effectively addresses the pathology. The target during the treatment was the Pcsk9 gene, associated with high cholesterol levels. After a month, Pcsk9 protein levels dropped to normal and remained low over 11 months of observation.
By the end of the experiment, scientists observed no signs of regression, suggesting the improvements could be stable over a long period. Further observations and experiments will reveal whether the effect can last for years.
Currently, dozens of different studies are being conducted that focus on epigenetic editing for the treatment or prevention of specific diseases. For example, a pilot clinical study targeting the MYC gene, which is overexpressed in many types of cancer, is already underway. If scientists manage to silence it, this could provide protection against cancer. Confirmation or refutation of this hypothesis will soon be obtained.
Recently, in another study, scientists discovered that epigenetic factors play a major role in the metastasis of cancer. It turns out that the impact on cells throughout life is more important than the genetic information encoded within them.
