Groundbreaking study paves the way for gene therapy in cancer and brain diseases - study
“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," researchers explained.
Researchers at the Technion Faculty of Biology have identified potential new innovative genetic therapies for cancer and brain diseases.
The findings were supported by the Israel Science Foundation (ISF), the US-Israel Binational Science Foundation (BSF), NSF-BSF Molecular and Cellular Biosciences, and the National Institutes of Health (NIH) and were published in the peer-reviewed journal Nucleic Acids Research.
The study was conducted in collaboration with the research group of Professor Heather Hundley from Indiana University and was led by Ph.D student Berta Eliad, master’s student Noa Schneider, and their advisor, Associate Professor Ayelet Lamn.
RNA editing system
The primary function of RNA is to create proteins through a biological process known as translation. In this process, RNA transmits genetic information that ribosomes translate into different proteins essential for cellular functions.
Our body uses an RNA editing system to make these proteins. RNA editing is a common post-transcriptional structure that is key for RNA function. RNA editing is a naturally occurring phenomenon in cells, but these researchers wanted to explore the possibility of directing these processes to repair mutated RNA.
The most frequent type of RNA editing is A-to-I RNA editing. This form of RNA editing is conducted by an enzyme called ADAR (Adenosine Deaminases Acting on RNA), altering one of the molecules within double-stranded RNA, converting it from adenosine (A) to inosine (I).
This seemingly minor modification has a huge role in human health; the alteration of editing occurs in many neurological diseases and cancers.
The results of the study
The research team used C. elegans (Caenorhabditis elegans) as the model organism for their study. C. elegans is a worm species that is commonly used in biological research because of its experimental amenability and body transparency.
The researchers examined the ADAR enzyme in C. elegans. According to a Technion press release on Thursday, November 21, the biologists pinpointed which RNA the ADAR enzyme preferentially edits and discovered that RNA editing occurs during the creation of new RNA molecules.
They also investigated how the localization of the ADAR enzyme affects its function. They identified that the ADAR enzyme is found near DNA molecules during cell division. Additionally, they discovered that in wild-type worms, the form of ADAR that edits coding and non-coding regions (ADAR2) is located in the nuclei and is adjacent to the chromosomes throughout all stages of the cell cycle.
The researchers found that ADAR is expressed in embryos, oocytes, and nerve cells but not in other cells, which proves that the mechanism is both tissue- and cell-type-specific.
The researchers believe that “this study provides new, groundbreaking insights in genetic medicine, which may lead to the development of innovative treatments for severe diseases.”
These new advancements in RNA research and gene therapy have transformative potential, offering new hope in the fight against cancer and neurological disorders and paving the way for innovative, personalized treatments that could reshape the future of medicine.
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