A new gene therapy technology mimicking the complex structure of viruses has been developed through joint research between South Korea and the United States.
A team of researchers led by Professor Lee Sang-min from the Department of Chemical Engineering at Pohang University of Science and Technology has developed a new therapeutic platform that mimics the complex and sophisticated structure of viruses using artificial intelligence (AI) in collaboration with Professor David Baker from the University of Washington, who won the Nobel Prize in Chemistry this year, the team noted on the 19th. The research was published in the international journal Nature on the same day.
Viruses have a unique structure that contains genes within a spherical protein shell, allowing them to replicate themselves. Recently, there has been active research using 'nanocages' that imitate the complex and sophisticated structure of viruses, effectively delivering therapeutic genes to target cells, as if the virus were attacking the host. However, existing nanocages were limited in the amount of genes they could hold due to their small size, and their simple structure posed challenges in implementing multiple functions like actual viral proteins.
To address this issue, the research team introduced AI-based computational design techniques. Although most viruses have symmetrical structures, there are slight misalignments. The research team used AI to design these structural features, creating various forms of nanocages, such as tetrahedrons, cubes, and dodecahedrons, for the first time in the world.
Analysis of the nanocages created using an electron microscope showed that the nanocages designed by AI exhibited accurate symmetrical structures as expected. The research team also confirmed that the nanocages containing genes successfully reached the target cells. In particular, the dodecahedron structure, which has a diameter of up to 75 nm (nanometers), is three times larger than existing gene delivery vehicles, allowing for significantly more genes to be contained.
Professor Lee Sang-min said, "The advancement of AI has opened an era where humanity can design and assemble artificial proteins as desired," and added, "I hope this research contributes to innovative advancements in various fields of medicine and life sciences, including gene therapeutics and next-generation vaccines."
Reference materials
Nature (2024), DOI: https://www.nature.com/articles/s41586-024-07814-1