RNA activation (RNAa), mediated by small activating RNAs (saRNAs), upregulates endogenous gene expression through recruitment of the Argonaute proteins to the promoter, natural antisense transcripts, or other noncoding RNAs. This mechanistically distinct modality addresses an unmet need, as few RNA therapeutics current...
Petra Sušjan-Leite, Peter Pečan, Ana Godeša et al.· Molecules· 0 citations
A unifying innate-sensing architecture for nucleic-acid therapeutics is defined and it is established that reactogenicity is not required for effective vaccination or gene delivery, enabling a broadly applicable strategy to widen the safety window of mRNA medicines, viral vectors, and in vivo genome editing.
D. Lainšček, Š. Malenšek, V. Forstnerič et al.· Molecular Therapy· 0 citations
This work fuses tamoxifen-responsive ER ligand-binding domains to CARs to generate ligand-inducible ON-switch CARs whose stability, signaling, and effector functions are precisely controlled by 4-hydroxytamoxifen.
Erik Rihtar, Tina Fink, Monika Belak et al.· bioRxiv· 0 citations
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