Regulation of gene expression via aptamer-mediated control of self-cleaving ribozymes
AssigneeMeiragtx UK II Limited
InventorsMichael J. Volles, Olivier F. Danos, Alex R. Boyne, Veronique Zennou, Xuecui Guo
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Abstract
The invention provides polynucleotide constructs for the regulation of gene expression by aptamer-based modulation of self-cleaving ribozymes and methods of using the constructs to regulate gene expression in response to the presence or absence of a ligand that binds the aptamer. The invention further provides methods for making and using riboswitches that decrease target gene expression in response to an aptamer ligand as well as riboswitches that increase target gene expression in response to an aptamer ligand.
Claims25
A polynucleotide construct for reducing the expression of a target gene in response to an aptamer ligand, the polynucleotide construct comprising (a) the target gene coding sequence flanked by a 5′ and a 3′ untranslated region (UTR);
- and (b) a riboswitch that comprises a twister ribozyme linked by a stem to an aptamer;
- wherein the aptamer comprises at least one stem;
- wherein the stem sequence linking the twister ribozyme to the aptamer comprises sequence that forms the aptamer stem and the P3 stem of the twister ribozyme;
- and wherein the target gene is linked to the P1 stem of the twister ribozyme;
- and wherein the riboswitch is located in (i) the 5′ UTR, (ii) the 3′ UTR, or (iii) the target gene coding sequence, 3′ of the start codon.
Dependencies are inferred from the claim text.