"Even if the lungs harden or the heart enlarges, they can be treated with genes."
Professor Kim Hyo-su of Seoul National University Hospital Biomedical Research Institute said in an interview at the Westin Josun Hotel in Jung District, Seoul, on the 6th, "DNA (deoxyribonucleic acid) and RNA (ribonucleic acid), and as delivery tools, genes can be inserted," adding, "We can expand the treatment scope beyond rare diseases to chronic diseases."
Only the necessary parts of DNA's genetic information are transferred to RNA to produce various proteins that govern life phenomena. Gene therapy is an approach that replaces a faulty gene with a normal one or alters gene function to treat genetic defects.
Kim said pulmonary fibrosis, in which the lungs harden, can be treated with genes. Pulmonary fibrosis is an intractable disease in which lung cells turn into fibrous tissue and cause breathing difficulties. Once lung tissue hardens, recovery is difficult and there is no way to prevent fibrosis.
Kim focused on the TIF1γ gene. An analysis of lung tissue from patients with pulmonary fibrosis found lower TIF1γ expression than in healthy people. When a TIF1γ gene therapy was administered to animals, it suppressed the progression of pulmonary fibrosis. The results were the same in experiments on human lung tissue cultured in vitro. Kim presented the findings in the international journal "Molecular Therapy" in Aug.
Kim believes heart failure, in which the heart becomes enlarged, and cirrhosis, in which the liver hardens and shrinks, can be treated in this way. Heart failure is a decline in cardiac function that causes fatigue and shortness of breath. Cirrhosis is a decline in liver function due to chronic inflammation. They share the common feature of organ tissue fibrosis.
"If the genes that regulate the heart muscle go wrong, the heart can become excessively enlarged, leading to heart failure and death," Kim said. "Because replacing the gene with a normal one can fundamentally cure the disease, it is an attractive therapy from the patient's perspective."
Kim graduated from Seoul National University College of Medicine and earned a master's and doctorate at the same graduate school. After serving as a professor at Seoul National University College of Medicine, Kim founded KimCell Engine in 2020. KimCell Engine aims to overcome intractable diseases by developing gene and cell therapies.
The pharmaceutical and biotech industry projects the global gene therapy market will grow from 36 trillion won last year to 141 trillion won by 2032. This is thanks to continued advances in gene therapy technology. A representative example is gene editing using CRISPR (CRISPR)-Cas9. When guide RNA recognizes and grips the DNA segment to be cut, the enzyme protein binds to the DNA and cuts it. Damaged genes can be excised and desired genes inserted.
There are also methods that do not directly modify DNA. These are RNA therapeutics that work like the COVID-19 vaccine. Delivering RNA copied from DNA information into cells leaves the patient's DNA intact while directly synthesizing the desired protein. Short microRNA can regulate the protein synthesis process. RNA therapeutics are expanding their application from cancer and genetic diseases to metabolic diseases such as diabetes and dyslipidemia.
CRISPR-Cas9 and RNA therapeutics are delivered into the human body using harmless viruses. Recently, technologies that do not use viruses have also been developed. They raise fewer safety concerns than virus-based gene therapies and are advantageous for mass production.
Gene therapies still have a way to go. "To advance gene therapy technologies, regulations must be eased," Kim said, adding, "We need systems like the U.S. Food and Drug Administration (FDA)'s regenerative medicine advanced therapy (RMAT)." RMAT designation can shorten the review and approval period for therapies.
References
Molecular Therapy (2025), DOI: https://doi.org/10.1016/j.ymthe.2025.08.035