KAIST Researchers Pioneer RNA Modification in Living Organisms

A research team led by Prof. Won Do Heo from the Department of Biological Sciences at Korea Advanced Institute of Science & Technology (KAIST) has developed a technology that allows selective RNA modification in living cells and animals. The findings of the study, published in Nature Chemical Biology, mark a step forward in RNA-based gene therapy and precision medicine.

KAIST researchers used CRISPR-Cas13, a system known for its ability to target RNA rather than DNA, to develop the world's first technology capable of selectively acetylating specific RNA molecules within the human body. The team developed a targeted RNA acetylation system called dCas13-eNAT10 to better understand and control how RNA works in cells. The study found that this system also helped export the RNA from the nucleus to the cytoplasm.

The study demonstrated the first successful in vivo RNA modification by successfully delivering the targeted RNA acetylation system in the liver cells of mice using adeno-associated virus (AAV) vectors. “The RNA chemical modification technology developed in this study can be widely used as an RNA-based therapeutic agent and a tool for regulating RNA functions in living organisms in the future,” Prof. Heo said.

For more information, read the article from KAIST.