Selectively expressed RNA molecules as a versatile tool for functionalized cell targeting

Frederik Rastfeld, Marco Hoffmann, Sylvie Krüger, Patrick Bohn, Anne-Sophie Gribling-Burrer, Laura Wagner, Nils Hersch, Carina Stegmayr, Lukas Lövenich, Sven Gerlach, Daniel Köninger, Christina Hoffmann, Helene L. Walter, Dirk Wiedermann, Hajaani Manoharan, Gereon R. Fink, Rudolf Merkel, Heribert Bohlen, Redmond P. Smyth, Maria A. Rueger and Bernd Hoffmann ()
Additional contact information
Frederik Rastfeld: Research Centre Juelich
Marco Hoffmann: Research Centre Juelich
Sylvie Krüger: Research Centre Juelich
Patrick Bohn: Helmholtz Centre for Infection Research
Anne-Sophie Gribling-Burrer: Helmholtz Centre for Infection Research
Laura Wagner: Research Centre Juelich
Nils Hersch: Research Centre Juelich
Carina Stegmayr: Research Centre Juelich
Lukas Lövenich: Research Centre Juelich
Sven Gerlach: Research Centre Juelich
Daniel Köninger: Research Centre Juelich
Christina Hoffmann: Research Centre Juelich
Helene L. Walter: Research Centre Juelich
Dirk Wiedermann: Multimodal Imaging Group
Hajaani Manoharan: Research Centre Juelich
Gereon R. Fink: Research Centre Juelich
Rudolf Merkel: Research Centre Juelich
Heribert Bohlen: SRTD biotech GmbH
Redmond P. Smyth: Helmholtz Centre for Infection Research
Maria A. Rueger: Research Centre Juelich
Bernd Hoffmann: Research Centre Juelich

Nature Communications, 2025, vol. 16, issue 1, 1-17

Abstract: Abstract Targeting of diseased cells is one of the most urgently needed prerequisites for a next generation of potent pharmaceuticals. Different approaches pursued fail mainly due to a lack of specific surface markers. Developing an RNA-based methodology, we can now ensure precise cell targeting combined with selective expression of effector proteins for therapy, diagnostics or cell steering. The specific combination of the molecular properties of antisense technology and mRNA therapy with functional RNA secondary structures allowed us to develop selectively expressed RNA molecules for medical applications. These seRNAs remain inactive in non-target cells and induce translation by partial degradation only in preselected cell types of interest. Cell specificity and type of functionalization are easily adaptable based on a modular system. In proof-of-concept studies we use seRNAs as platform technology for highly selective cell targeting. We effectively treat breast tumor cell clusters in mixed cell systems and shrink early U87 glioblastoma cell clusters in the brain of male mice without detectable side effects. Our data open up potential avenues for various therapeutic applications.

Date: 2025
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DOI: 10.1038/s41467-024-55547-6

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