This article has been reviewed according to Science X's editorial process and policies. Editors have highlighted the following attributes while ensuring the content's credibility: RNA-based therapeutics use RNA as a blueprint that enables cells to produce specific proteins—including proteins that can precisely modify genes. For this to work, the RNA must reach the inside of the cell intact.
Delivery systems currently used for this purpose include virus-derived vehicles and lipid nanoparticles, tiny particles made of fat-like molecules. Both approaches have limitations. Researchers are therefore working on new mechanisms that can deliver RNA into cells more efficiently and, in the future, more selectively.
Research teams at the Institute of Stem Cell Research (ISF) and the Institute of Developmental Genetics (IDG) at Helmholtz Munich and the Technical University of Munich have constructed an RNA transporter from the ground up. The researchers combined functional protein building blocks with a structural protein designed using generative AI. This protein forms the scaffold of the vehicle and can adopt shapes that do not occur in nature.
"We did not want to recreate nature, but to design new structures for a specific task: the efficient delivery of RNA," says Dr. Christoph Gruber, team leader at the ISF and co-first author of the study. The study is published in the journal Nature.
The team tested more than 100 variants. Surprisingly, protein structures with non-natural geometries performed particularly well. STV-C8 was the most efficient.
"The fact that a structure that differs so markedly from natural viral capsids works particularly well was a key finding for us," says Dr. Maren Kirstin Schuhmacher, postdoctoral researcher at the ISF and also co-first author. "It demonstrates the potential of using AI to systematically expand the protein design space." In cell culture, STV-C8 delivered RNA into target cells far more efficiently than the virus-like particles and lipid nanoparticles tested.
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