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.
Claims23
A polynucleotide cassette for inducing expression of a target gene in response to an aptamer ligand, the polynucleotide cassette comprising a riboswitch that comprises an aptamer that is located 3′ or 5′ of a twister ribozyme, wherein the aptamer comprises at least one stem, wherein the P1 stem of the twister ribozyme and the stem of the aptamer comprise an alternatively shared stem arm sequence, and wherein:
- (a) when the aptamer is located 3′ of the twister ribozyme, the alternatively shared stem arm sequence is capable of alternatively forming all or part of (i) the 3′ arm of the twister ribozyme P1 stem or (ii) the 5′ arm of the stem of the aptamer, and
- (b) when the aptamer is located 5′ of the twister ribozyme, the alternatively shared stem arm sequence is capable of alternatively forming all or part of (i) the 5′ arm of the twister ribozyme P1 stem or (ii) the 3′ arm of the stem of the aptamer.
Dependencies are inferred from the claim text.