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US 2026/0224679 A1Published application
SWITCHABLE mRNA EXPRESSION PLATFORM
OwnerPetr SULC, Mark KNAPPENBERGER +3inventors
InventorsPetr SULC, Mark KNAPPENBERGER, Archana KIKLA, Matthen SAMPLE, Karen ANDERSON
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Abstract
Described herein is a gene delivery system exhibiting high stability, compactness, and structural utility. The delivery system has a built-in ON switch, which only expresses the gene only in cells that contain a specified “trigger” RNA molecule, which then enables the translation of the delivered construct.
Claims20
A nanoparticle composition comprising a folded single-stranded nucleic acid nanostructure comprising:
- a 5′-untranslated region (5′-UTR);
- a coding sequence;
- a structuring sequence that is complementary to the coding sequence and the 5′-UTR or a region of the coding sequence and the 5′-UTR;
- a 3′-untranslated region (3′-UTR);
- and
- a polyadenosine (poly-A) tail;
- wherein the structuring sequence is bound to the 5′-UTR and encapsulated in the nanostructure.
4 more elements
A method for selectively expressing a coding sequence in a target cell, the method comprising:
- delivering to a target cell a nanoparticle composition comprising a folded single-stranded nucleic acid nanostructure comprising:
- a 5′-untranslated region (UTR);
- a coding sequence;
- a structuring sequence that is complementary to the coding sequence and 5′-UTR or a region of the coding sequence and the 5′-UTR;
- a 3′-UTR;
- and
- a polyadenosine (poly-A) tail;
- wherein the structuring sequence is bound to the 5′-UTR and encapsulated in the nanostructure;
- wherein upon delivering the nanoparticle composition to the target cell, a target cell nucleic acid molecule binds the structuring sequence and displaces the 5′-UTR, thereby releasing the 5′-UTR for translation and expression of the coding sequence.
6 more elements
Dependencies are inferred from the claim text.