△ Professor Kim Young-kwang, Department of Pathology, The Catholic University of Korea College of Medicine, and Professor Kim Ki-pyo, Department of Biomedical Sciences

A noteworthy research on treating intractable neurological diseases is beginning at The Catholic University of Korea Catholic Medical Center, which operates the largest medical network in Korea.

Professor Kim Young-kwang's research team (Department of Pathology, The Catholic University of Korea College of Medicine) from the Synthetic Biology Project Group of the Basic Medical Science Project Promotion Team at The Catholic University of Korea Catholic Medical Center has officially been selected by the government for the 'Next-Generation RNA Gene Editing Technology' development project and has begun full-scale research.

This research is part of the 2025 Bio-Medical Technology Development Project organized by the Ministry of Science and ICT, aiming to develop new gene editing technology that can precisely edit and regulate RNA (ribonucleic acid) and apply it to the treatment of intractable neurological diseases.

Professor Kim Young-kwang has been selected as the principal investigator for this project, with Professor Kim Ki-pyo (Department of Biomedical Sciences, The Catholic University of Korea College of Medicine), Professor Chae Dong-woo (Yonsei University College of Medicine), and CEO Song Ji-hwan (IPS Bio) participating as co-researchers. The research will be conducted over the next five years, receiving a total of 2.8 billion KRW in government support, of which 1.86 billion KRW will be executed through The Catholic University of Korea Industry-Academic Cooperation Foundation.

The core technology in this research is the 'RNA gene scissors.' Simply put, gene scissors are technology that accurately cuts and changes genetic information (DNA or RNA) in our bodies. Most widely known gene scissors to date have targeted DNA. However, DNA editing results are irreversible, making recovery difficult in case of side effects, and there have been safety concerns about in vivo gene correction due to cellular toxicity that occurs during the DNA cutting process.

In contrast, RNA is a 'copy' made by DNA, which is the information used to make actual proteins. Editing RNA allows for the regulation of disease-related protein production while leaving DNA intact, making it a much safer and more precise treatment method.

RNA-targeted gene scissors technology is one of the most notable technologies in the field of life sciences recently. In particular, the key to this project is to create RNA gene scissors that are much more precise and efficient than existing ones.

The title of this project is 'Advanced Next-Generation RNA-Targeted CRISPR Gene Scissors Using Directed Evolution and Application to Intractable Neurological Disease Treatment.' Here, 'directed evolution' refers to a technology that rapidly reproduces in the laboratory the evolutionary process that takes place over millions of years in nature to create superior proteins.

Directed evolution is a technology that won the 2018 Nobel Prize in Chemistry, and CRISPR gene scissors are a technology that won the 2020 Nobel Prize in Chemistry, now causing innovation in various fields such as genetics, cancer treatment, and rare disease research. Professor Kim Young-kwang's team is taking on the challenge of creating a new gene scissors that can regulate RNA more accurately and safely by combining these two technologies.

Despite the high efficiency of existing RNA gene scissors, concerns about target accuracy and cellular toxicity have not been resolved, limiting their application as therapeutics. The research team plans to challenge the creation of new gene scissors through directed evolution experiments and protein structure prediction AI technology that can simultaneously improve therapeutic effects and safety.

This research is particularly noteworthy because it is directly linked to the treatment of 'intractable neurological diseases.' Intractable neurological diseases are illnesses that occur due to abnormalities in the brain, spinal cord, and nervous system, with representative examples including amyotrophic lateral sclerosis (ALS), Huntington's disease, Parkinson's disease, and rare genetic disorders. Currently, these diseases are difficult to cure completely, and symptoms often worsen progressively, making the development of treatments urgent.

Professor Kim Young-kwang's team is developing methods to identify RNA that causes diseases using RNA gene scissors technology, and to regulate or edit it to enhance therapeutic effects. They also plan to attempt new treatment strategies using RNA gene scissors for intractable neurological diseases such as Huntington's disease and Pelizaeus-Merzbacher disease. When this technology is completed in the future, it will be able to present new treatment strategies for diseases that were previously untreatable.

Professor Kim Young-kwang also worked as a neurology specialist in clinical practice for five years. However, he turned to basic medical science research to go beyond direct patient treatment and study the root causes of diseases and create new treatments. He has since been continuously researching CRISPR gene scissors and RNA editing technology, and this project has become an opportunity to open a more full-scale research field based on accumulated experience and technical skills. This research, which incorporates both clinical experience as a physician and scientific insight as a researcher, is also significant in that it is an applied research that can be connected to actual patient treatment, not just theory.

This project is being conducted as a multidisciplinary research collaboration among professors from various fields within The Catholic University of Korea College of Medicine, including pathology and biomedical sciences, as well as external universities and private companies. This means comprehensive research that encompasses not only single technology development but also the development of new drug candidates, experimental model verification, and industrialization possibilities.

This project does not simply stop at advancing gene scissors technology. It is expected to be an important stepping stone for opening the era of 'precision medicine' to protect human health and life. Precision medicine refers to providing customized treatment according to each patient's genetic information. RNA gene scissors technology is one of the core technologies of such precision medicine and is expected to be widely used in the treatment of various diseases such as cancer, genetic diseases, and degenerative diseases in the future.

Professor Kim Young-kwang, who is leading this project as part of the Synthetic Biology Project Group of The Catholic University of Korea Catholic Medical Center's Basic Medical Science Project Promotion Team, stated, "I hope this research can give hope to many patients suffering from diseases without treatment options," adding, "I will prioritize the development of accurate and safe medical technology."

△ Schematic diagram of next-generation RNA gene editing technology development using directed evolution