Kim Sun-wook, director of the Future Animal Resources Center at the Korea Research Institute of Bioscience and Biotechnology (KRIBB), and his research team announced on the 28th that they have developed a new treatment method that precisely suppresses the cause of progeria using next-generation gene regulation technology, marking the first such development in the world. The research results were published online in the international journal "Molecular Therapy" in June.
Progeria (Hutchinson-Gilford Progeria Syndrome, HGPS) is a rare and intractable genetic disorder that occurs in 1 in about 8 million people. Children with progeria experience wrinkling of the skin and stunted growth after 1 to 2 years of age, with their bones and blood vessels aging rapidly. The average life expectancy is only about 14.5 years, and there is currently no treatment for a complete cure.
The research team focused on the progerin gene, which is the cause of progeria. Progeria occurs due to a single mutation in the LMNA gene. This mutation produces an abnormal protein called "progerin" within cells, which damages the nuclear structure of cells and accelerates aging, leading to weakened bones and hardened blood vessels as seen in the elderly, ultimately causing the failure of major organ functions.
The research team created RNA scissors that accurately distinguish and target the progerin gene from normal genes. These RNA scissors can precisely eliminate only the protein that causes the disease without affecting normal proteins. Notably, this technology is much safer than existing gene editing technologies because it regulates only RNA without altering deoxyribonucleic acid (DNA).
When the RNA therapy was applied to a mouse model with the progeria gene, symptoms such as hair loss, skin atrophy, spinal deformities, and decreased motor abilities showed significant improvement. Additionally, body weight increased, reproductive organ function was recovered, and even heart and muscle functions were restored, showing characteristics closer to those of healthy rats.
Beyond a simple disease treatment technology, the possibility of precisely regulating the fundamental causes of aging has also been confirmed. In fact, the research team observed that progerin gradually increases in the skin cells of older individuals and found that the application of this RNA scissors technology also partially suppresses natural aging phenomena, according to experimental results.
Director Kim Sun-wook noted, "This technology can be applied to more than 15% of other genetic disorders caused by RNA editing errors, in addition to progeria," and added, "I expect this to become a platform technology that can be expanded to age-related diseases, cancer, and neurodegenerative diseases in the future."
References
Molecular Therapy (2025), DOI: https://doi.org/10.1016/j.ymthe.2025.06.017