Israeli Study Could Lead to Gene Therapy for Cancer, Brain Diseases

Researchers at the Technion Faculty of Biology have presented findings that could lead to new genetic treatments for cancer and brain diseases. Published in the journal Nucleic Acids Research, the study was led by PhD student Berta Eliad, Master’s student Noa Schneider, and their advisor, Associate Professor Ayelet Lamm and was a collaboration with the research group of Professor Heather Hundley from Indiana University.

DNA is effectively the body’s instruction manual for producing proteins, whereas RNA is like a copy of one page from the manual — a recipe for making a specific protein. To make more extensive, more diverse sets of these “protein recipes,” our body uses an RNA editing mechanism. This process changes the RNA sequence, resulting in a changed recipe.

RNA editing occurs naturally in cells, but researchers are currently exploring the possibility of directing these processes to repair mutated RNA.

One of the most common types of RNA editing found in nature is A-to-I RNA editing. In this process, an enzyme called ADAR changes one of the molecules that make up the RNA, converting it from adenosine (A) to inosine (I). Disruptions in this process can lead to cancer, neurodegenerative disease, and misactivation of the immune system.

The Technion researchers examined the ADAR enzyme in C. elegans — a worm commonly used as a model organism in biological research due to its transparency and rapid development. The researchers discovered that in these worms, the ADAR enzyme is found near DNA molecules during cell division and concluded that RNA editing occurs when new RNAs are created. They also discovered that ADAR is expressed in embryos, oocytes and nerve cells, but not in sperm cells or other cells, meaning the mechanism is selective for specific tissues. Furthermore, they discovered a protein that controls the location of the ADAR enzyme within the cell and identified which RNA the ADAR enzyme “prefers” to edit.

“Our findings show where RNA editing takes place and which factors regulate it, allowing us to understand how RNA editing can be used to repair damaged genes,” the researchers said.

“This study provides new, groundbreaking insights in genetic medicine, which may lead to the development of innovative treatments for severe diseases.”

The study was supported by the Israel Science Foundation (ISF), the US-Israel Binational Science Foundation (BSF), NSF-BSF Molecular and Cellular Biosciences, and NIH.