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Survival - in the brain

In addition to their well-known role, RNA molecules supervise various actions throughout the cell, a role previously associated only with proteins

Stephen Hawking at a dinner at Tel Aviv University in 2006. Photo: Danor Aharon / Shutterstock.com
Stephen Hawking at a dinner at Tel Aviv University in 2006. Photo: Danor Aharon / Shutterstock.com

In the past, the plot of the story of the biological control processes in the living cell was considered relatively simple: the operating instructions of the body were encoded in DNA and passed from generation to generation; The RNA functioned as a messenger that delivered the instructions and led to the creation of proteins; And the proteins performed the day-to-day operations in addition to special operations. But in the last decade - after it was discovered that in addition to their well-known role, RNA molecules supervise various actions throughout the cell, a role previously associated only with proteins - it is being written in this plot.

Dr. Eran Hornstein, from the department of molecular genetics at the Weizmann Institute of Science, studies microRNA molecules (which, as you can learn from Shaman, are very short RNA molecules). Dr. Hornstein's research shows that microRNA plays a crucial role in supervising the normal functioning of the cell: this is the reason why defects or malfunctions in the genes encoding microRNA may contribute to the development of diseases. For example, Dr. Hornstein's research group discovered that some of the microRNA molecules play a crucial role in the survival of motor nerve cells.

When these molecules fail to perform their function, the result may be the development of amyotrophic lateral sclerosis (ALS), a destructive disease of the motor nerve cells, which gradually deprives the patient of the ability to move. Dr. Hornstein's research illustrates the involvement of an enzyme called "Dicer" in the changes that occur in the disease. This enzyme is responsible for processing most of the microRNA molecules, so that they can develop into their mature and active form. "When 'Deicer' stops working," says Dr. Hornstein, "severe defects are created in the maturation of the microRNA molecules, and as a result they do not work in the nerve cells." His research showed that a malfunction in the "Deicer" activity or in the function of the microRNA molecules could cause the death of nerve cells and the degeneration of the muscles they control.

Dr. Eran Hornstein and Dr. Anna Ameda. Let him write the plot. Photo: Weizmann Institute
Dr. Eran Hornstein and Dr. Anna Ameda. Let him write the plot. Photo: Weizmann Institute

The results of the experiment that Dr. Hornstein and his group members performed on human samples and mouse models showed that finding ways to increase the activity of the "Deicer" enzyme may, perhaps, lead to the treatment of ALS, which is considered an incurable disease.

In a study led by Dr. Anna Ameda, a physician and research student in Dr. Hornstein's laboratory, the members of the laboratory tested a well-known drug for urinary tract infections called "Enoxacin", which was previously reported to increase the activity of "Deicer". The experiments carried out by the scientists indicate that enoxacin may indeed slow down the deterioration of the condition of ALS patients. Since this drug is well known, Dr. Hornstein says that the road to its approval for a clinical trial in diseases like ALS should be relatively short.

In addition to this research, Dr. Hornstein and his team are working on research that is designed to allow them to understand how microRNA molecules work in other vital cells in the body, and they also strive to develop additional drugs based on RNA.

"I hope," says Dr. Hornstein, "that understanding the ways of action of tiny molecules of the microRNA type will advance us to our goal - understanding the molecular mechanisms that control the activity of genes, and how they affect the normal functioning of cells and tissues." At the same time, we hope that our basic observations will contribute to the ability to develop a medicinal solution for incurable diseases".

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