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Compugen announces a scientific breakthrough: a fifth nucleic acid in RNA is more common than known

The company's scientists discovered that a fifth nucleic acid appears in thousands of sites in human RNA, and contributes to a large number of possible combinations in RNA sequences * The discovery is published in the scientific journal Nature Biotechnology

Compugen announces a scientific breakthrough. The company's scientists have identified thousands of sites where a fifth nucleic acid (Inosine, I) appears in human RNA (RNA - a stage in the process of coding genes for proteins) in addition to the four known nucleic acids in RNA - A, U, G and - C. The presence of The fifth nucleic acid causes significant changes in the sequence of nucleic acids in the human transcriptome, which is the collection of all human RNA sequences bridging the genome (the collection of all genes) and the proteome (the collection of all proteins). The company has identified more than 10,000 sites where a change in the genetic code occurs as a result of a phenomenon called RNA Editing, where the nucleic acid inosine (I) replaces the nucleic acid adenosine (A). Until today the phenomenon was considered rare and about 100 such cases were known. Since the editing phenomenon is done after the transcription from the DNA, this means that the range of possible combinations of RNA sequences is much wider than was known until now from the genomic information. The large number of sites found makes it possible to reveal features of these sites, and provides initial clues to their function. The discovery is published in the scientific journal Nature Biotechnology.

RNA editing is a modification of the RNA sequence in which a change in the nucleic acids occurs after the DNA is transcribed into RNA. It is known that RNA editing is an essential element in the development of mammals, and evidence has been published indicating that the phenomenon may be widespread, but only a few sites of RNA editing were known until now. Before the present discovery, the general opinion was that it was not possible to systematically discover a large number of such sites using the existing experimental methods and computational tools.

Defects in the process of converting nucleic acids A to I have been associated with several neurological diseases such as muscular dystrophy (ALS), brain tumors (gliomas), epilepsy, and depression. Most of the consequences of RNA editing are still unknown, but it is widely believed that the phenomenon may play a significant role in the expression and regulation of genes.

In the study published today, Compugen scientists discovered 12,723 sites of RNA editing in 1637 genes by using the computational models and prediction systems developed by the company. In selected cases, confirmation experiments were also performed in Compugen's molecular biology laboratories, as well as in the laboratories of the Department of Cell Biology and Genetics at the University of Vienna, Austria. The research was done in collaboration with Professor Gidi Ravavi, head of the Cancer Research Center at the Sheba Medical Center at Tel Aviv University.

"Today's announcement is further evidence of Compugen's unique predictive approach and the value of combining ideas and methodologies from the fields of mathematics, computer science and physics with the life sciences," said Dr. Mor Amitai, Compugen's president and CEO. "Compugen's pioneering research has already led to a deeper understanding of important biological phenomena such as alternative splicing - the formation of several proteins from one gene; antisense - transcription of the two complementary strands of DNA from the same place in the genome; And now RNA editing is used as an important natural mechanism in the modification of RNA sequences after expression. Compugen uses this understanding for the purpose of discoveries for the purposes of curing and diagnosing diseases and to continue improving its predictive models, thus increasing its chances of further discoveries in the future by using these means."

Compugen's LEADS platform

The results announced today regarding Compugen's ability to systematically identify a large number of sites that have undergone A to I RNA editing, and to identify their exact location in the human genome, are based on the use of LEADS, a platform for computational biology. The LEADS system uses unique algorithms to predict and represent the transcriptome of complex organisms. The LEADS platform is used to decipher and predict genes, transcripts, gene variants (splice variants) and proteins and to discover information about their function. This platform cultivates important biological phenomena and provides Compugen and its customers with solutions to complicated biological challenges with the aim of accelerating the development of drugs and diagnostic markers. With the help of the LEADS platform, Compugen identified thousands of new genes and proteins, some of which have therapeutic potential.

About Compugen

Compugen, a drug discovery and diagnostic marker company, increases the chances of success of developing innovative drugs and diagnostic products by integrating ideas and research methods from the fields of mathematics, computer science and physics into the life sciences.

The product of this unique combination is predictive models and drug discovery engines that advance the understanding of important biological phenomena, as well as enable the discovery of potential therapeutic products and diagnostic markers. Several proteins with therapeutic potential discovered by Compugen are in initial stages of development by the company. The company has collaborations with leading pharmaceutical and diagnostics companies, such as: Novartis, Abbott Laboratories and Diagnostics Product Corporation.

CompuGen's headquarters, research center, and laboratories are located in Tel Aviv and its business development center is located in Silicon Valley, California. Compugen has subsidiaries: Kedem Biosciences - a company for the discovery of small molecules for pharmaceutical purposes, and Avogen - a company in the field of agricultural biotechnology.

The company employs about 130 employees, about 100 of whom are mathematicians, engineers, scientists and doctors who make up one of the largest teams in the world for developing algorithms and models for deciphering life processes. More information can be obtained on the company's website:

For the electronic version of the article in the journal Nature Biotechnology

The human genome scientist

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