Seemingly small in size but big in impact, miRNAs are quietly revolutionizing the biological world. These molecules, which are at the root of many diseases by controlling the expression of genes, are opening the doors to new treatment and diagnosis methods in the medical world. Perhaps in the near future, we will be able to fight many diseases much more effectively thanks to microRNA-based therapies!

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microRNA: The Hidden Rulers of Cells and New Hope for Fighting Disease

It is well known that structures such as DNA and mRNA, which carry genetic information, are behind many of the complex events that take place in our cells. However, microRNA (miRNA), one of the hidden heroes of biology, has a key role in regulating all these processes! miRNAs, which are not talked about much but accomplish great things, almost secretly manage the control center of the cell. Thanks to Victor Ambros and Gary Ruvkun, who received the 2024 Nobel Prize for their work on microRNAs, interest in microRNAs has increased. Let’s get to know these small but effective molecules briefly!

microRNA What is it?

Briefly defined, microRNAs (miRNAs) are small RNA molecules, approximately 22 nucleotides in length, that are located inside the cell. They do not code for proteins but indirectly control protein production. How? miRNAs bind to mRNAs carrying genetic messages and prevent them from doing their job. In a way, they determine when genes are active and how much protein is produced. It is hard not to be surprised to learn that they have such a say in the functioning of the cell!

microRNA What does it do?

Here’s the kicker: miRNAs bind to mRNA and either degrade and destroy it or stop protein synthesis. This is a critical step to control gene expression and maintain balance in the cell. They even regulate many biological processes by influencing the behavior of the cell. These processes range from cell division to death.

In other words, these small molecules act like a “control panel” inside the cell, regulating which genes are “on” or “off”. This is exactly why miRNAs are referred to as “silent rulers” in the biological world.

microRNA and Diseases: The Hidden Link in the World of Health

The gene regulatory role of microRNAs places them at the center of diseases. In particular, miRNAs have been strongly associated with many diseases such as cancer, heart disease, neurodegenerative disorders (e.g. Alzheimer’s) and metabolic diseases. Here are a few striking examples:

Cancer: miRNAs can often be either overactive or completely repressed in cancerous cells. Some miRNAs function as tumor suppressors, while others may act as oncogenes (cancer-forming). Therefore, analyzing miRNA profiles becomes crucial in diagnosing cancer and monitoring response to treatment.
Heart Disease: Certain miRNAs play important roles in conditions such as healing damage after a heart attack. Events such as the growth or death of heart muscle cells are complex processes governed by miRNAs.
Neurological Diseases: In diseases such as Alzheimer’s and Parkinson’s, miRNAs can affect the function of brain cells. Research shows that some miRNAs play a role in maintaining or disrupting the health of nerve cells.

miRNA in the Future: New Horizons in Treatment and Diagnosis

This controlling role of microRNAs in cells makes them a powerful tool in the medical field. miRNA-based therapies are promising in the prevention or treatment of many diseases. For example, studies on suppressing or activating target genes in cancer treatment with the help of miRNAs continue rapidly.

MicroRNA Inhibitors: If a miRNA is overactive and causes disease, inhibitors can be developed to stop this miRNA. This prevents the suppression of disease-causing genes.
miRNA Supplements: On the other hand, the balance in the cell can be restored by replacing missing or inadequately functioning miRNAs with synthetic miRNAs produced in the laboratory.

miRNAs can also be used as biomarkers in complex diseases such as cancer. Measuring the levels of specific miRNAs in blood tests has great potential for early diagnosis and monitoring disease progression.

miRNA and Personalized Medicine

In the field of personalized medicine, which we have heard of frequently in recent years, miRNAs are in the lead role! By analyzing the genetic and miRNA profiles of patients, it becomes possible to tailor treatment options to the individual. Especially in cancer treatment, more targeted and effective treatments are expected to be developed thanks to these personalized approaches.