Patent ID: 8603996
Filing Date: 2013-12-10
Classification: A61P,C12N

Abstract:
1. An aptamer-regulated ribozyme for cleaving a target nucleic acid, comprising (a) a trans-acting hammerhead ribozyme comprising catalytic core, a 5′ targeting arm which hybridizes to a 3′ sequence of said target nucleic acid, a 3′ targeting arm which hybridizes to a 5′ sequence of said target nucleic acid, and a stem duplex region extending from said catalytic core with a single-stranded loop region opposite to said catalytic core; (b) an information transmission domain (ITD) having a first end and a second end, which information transmission domain is directly coupled to said loop through said first end; and (c) an aptamer coupled to said information transmission domain through said second end, said aptamer binds to a ligand, wherein said ITD is between the trans-acting hammerhead ribozyme and the aptamer, and comprises: wherein binding of said ligand to said aptamer favors a conformation change in the aptamer, wherein said conformation change causes the ITD to favor the binding of the general transmission region to said competing strand via a strand-displacement mechanism; and, wherein hybridization between the general transmission region and the competing strand causes, via the interaction of said information transmission domain with one or more of said loop, said stem or said catalytic core, said aptamer-regulated ribozyme to cleave the target nucleic acid at a rate dependent upon the presence or absence of said ligand.