Seoul National University Hospital Strengthens Joint Research on Rare Disease Gene Therapy with Nobel Laureate
- Consulting with Professor Craig Mello through collaboration with the Department of Genetic and Cellular Medicine at UMass Chan Medical School
- Discussion on strategies for developing RNA and gene therapies for rare diseases and plans for clinical application
[Photo 1] Craig Mello, Distinguished Professor at UMass Chan Medical School (second from left), Jong-Hee Chae, Director of Seoul National University Children's Hospital (third from left), and others posing for a commemorative photo.
[Photo 2] An advisory meeting on the development of RNA and gene therapies for rare diseases, held on the 11th.
The Rare Disease Center at Seoul National University Hospital (Director Byung-Chan Lim) is strengthening joint research on RNA and gene therapies for rare diseases with Craig C. Mello, Distinguished Professor at the University of Massachusetts Chan Medical School (UMass Chan Medical School), who received the 2006 Nobel Prize in Physiology or Medicine for the discovery of RNA interference (RNAi).
During an advisory meeting held on the September 11th, Professor Mello and the research team discussed strategies for developing RNA and gene therapies for rare diseases, plans for clinical application, and directions for patient-tailored treatment research. Building on their existing partnership, both sides plan to expand their research into therapeutic treatments following this meeting.
This collaboration builds upon a Memorandum of Understanding (MOU) signed last May between Seoul National University Hospital and the Department of Genetic and Cellular Medicine at UMass Chan Medical School regarding the development of gene therapies for rare diseases and personnel exchange. The two institutions have agreed to pursue the joint development of gene and cell therapies, conduct clinical research, and facilitate researcher exchanges, focusing on severe neurogenetic disorders such as neurodevelopmental disorders, epileptic encephalopathy, and neuromuscular diseases.
Professor Mello discovered RNA interference (RNAi) alongside Professor Andrew Z. Fire. RNAi is a mechanism that suppresses the expression of specific genes; it is widely used in gene function research and has served as the scientific foundation for the development of siRNA-based therapeutics. In addition to studying RNAi and gene regulation mechanisms in germ cells, Professor Mello is actively involved in translating basic research findings into the development of therapeutic treatments.
The Rare Disease Center at Seoul National University Hospital supports patients with hard-to-diagnose rare diseases through precision diagnostics—including genomic testing—and multidisciplinary care. Recently, the center has expanded its scope to include research into therapeutic technologies that directly target the root causes of diseases, such as RNA and gene therapies.
Jong-Hee Chae, Director of Seoul National University Children's Hospital, stated, "In the field of rare diseases, developing practical treatments that can be offered to patients following an accurate diagnosis is of paramount importance." He added, "We intend to use this advisory meeting with Professor Mello—a pioneer in RNA interference research—as an opportunity to advance our research into patient-specific RNA and gene therapies and to further expand our collaboration with global research institutions."