Seoul National University Hospital Expands Research Collaboration on RNA and Gene Therapies for Rare Diseases with Nobel Laureate

Building on an Existing Collaboration with the University of Massachusetts Chan Medical School
Advisory Meeting with Nobel Laureate Craig Mello
Discussing Strategies for the Development and Clinical Application of RNA and Gene Therapies for Rare Diseases

The Rare Disease Center at Seoul National University Hospital is expanding its research collaboration on RNA and gene therapies for rare diseases with Craig Mello, Distinguished Professor at the University of Massachusetts Chan Medical School, who was awarded the Nobel Prize in Physiology or Medicine in 2006 for his discovery of RNA interference (RNAi).

The Rare Disease Center announced on the 29th that it had held an advisory meeting with Professor Mello on the 11th. During the meeting, they discussed strategies for developing RNA and gene therapies for rare diseases, plans for clinical applications, and directions for personalized therapy research. With this advisory meeting as a turning point, Seoul National University Hospital plans to further expand its research on therapeutics for rare diseases based on its existing collaboration with Professor Mello.

Participants are speaking at the advisory meeting on the development of RNA and gene therapies for rare diseases held at Seoul National University Hospital on the 11th. Seoul National University Hospital

This collaboration is based on the memorandum of understanding (MOU) signed in May of last year between Seoul National University Hospital and the Department of Gene and Cell Medicine at the University of Massachusetts Chan Medical School, covering the development of gene therapies for rare diseases and personnel exchange. The two institutions agreed to jointly develop gene and cell therapies, conduct clinical research, and promote researcher exchange for severe neurogenetic disorders, such as neurodevelopmental disorders, epileptic encephalopathies, and neuromuscular diseases.

Professor Mello, together with Professor Andrew Fire, was co-awarded the Nobel Prize in Physiology or Medicine in 2006 for the discovery of RNA interference (RNAi). RNAi is a mechanism that selectively suppresses the expression of specific genes and has been widely used in functional genomics research. It has formed the scientific foundation for the development of small interfering RNA (siRNA)-based therapeutics.

Professor Mello has conducted extensive research on RNAi and the mechanisms of gene regulation in germ cells. He is also engaged in projects bridging basic research achievements and the development of therapeutics. Seoul National University Hospital discussed with Professor Mello various strategies for the development and clinical application of RNA and gene therapies for rare diseases, utilizing his research expertise.

The Rare Disease Center at Seoul National University Hospital supports patients with difficult-to-diagnose rare diseases through precision diagnosis, including genome analysis, and multidisciplinary care. Recently, the center has expanded its research scope to include new therapeutic technologies, such as RNA and gene therapies that directly target the causes of diseases.

Chae Jonghee, President of Seoul National University Children's Hospital, stated, "It is essential in the field of rare diseases to not only provide an accurate diagnosis, but also to develop effective treatment methods that can be offered after diagnosis. Building on Professor Mello's pioneering work in RNA interference, we will advance the research and development of personalized RNA and gene therapies and further expand collaborations with global research institutions."

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