AI-designed proteins enable a new generation of RNA transporters
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 at Helmholtz Munich and the Technical University of Munich have developed an RNA transporter engineered using generative AI. The synthetic protein structure, named STV-C8, surpasses traditional delivery systems like virus-like particles and lipid nanoparticles in efficiency. Tests in cell cultures showed STV-C8 performed better, requiring less RNA and delivering targeted cargo.
Mice studies demonstrated safe lung expression without toxicity or immune issues. In pig models, STV-C8, loaded with CRISPR/Cas9 components, successfully removed a segment from the dystrophin gene associated with Duchenne muscular dystrophy. While still experimental, this bottom-up RNA transfer platform could revolutionize therapeutic applications once targeting and distribution mechanisms are further evaluated.
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