Regulation of gene expression through aptamer-modulated polyadenylation
AssigneeMeiragtx UK II Limited
InventorsMichael J. Volles, Olivier F. Danos, Xuecui Guo
Life of this patent
Estimating the term…
Abstract
The invention provides polynucleotide constructs for the regulation of gene expression by aptamer-based modulation of U1 small nuclear ribonucleoprotein (snRNP)-mediated suppression of polyadenylation 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 polynucleotide construct contains a U1 binding site in the context of a riboswitch comprising an effector region and an aptamer such that when the aptamer binds a ligand, target gene expression occurs.
Claims30
A polynucleotide construct for the regulation of the expression of a target gene comprising a polynucleotide sequence comprising the target gene coding sequence and a 3′ untranslated region (UTR) comprising a polyadenylation signal, and a riboswitch, wherein the riboswitch comprises an effector region and an aptamer, wherein the effector region comprises a stem-forming sequence that is 8 to 11 base pairs comprising a U1 snRNP binding site and sequence complementary to the U1 snRNP binding site, wherein the riboswitch is located in the 3′ UTR of the target gene 5′ of the polyadenylation signal.
Dependencies are inferred from the claim text.