Back to results
US 6,713,456 B1Granted
Nucleozymes
AssigneesMassachusetts Institute of Technology, University of Montreal
InventorsNassim Usman, Robert J. Cedergren, Jean-Pierre Perreault, Jing-Hua Yang, Alexander Rich
Life of this patent
Estimating the term…
Abstract
Nucleozymes containing ribonucleotides and deoxyribonucleotides or nucleic acid analogues are described herein. The nucleozymes have catalytic activity and are significantly more resistant to degradation than their all-RNA ribozyme counterparts. Also described are methods for preparing the nucleozymes along with methods of using nucleozymes, e.g., as therapeutic agents.
Claims4
A pharmaceutical composition, comprising:
- at least one enzymatic nucleic acid molecule having a ribonucleotide at a catalytically critical site, at least one deoxyribonucleotide and at least one nucleic acid analog;
- and
- a pharmaceutically acceptable carrier.
A pharmaceutical composition, comprising:
- at least one enzymatic nucleic acid molecule having a ribonucleotide at a catalytically critical site and at least one deoxyribonucleotide or nucleic acid analog and has enzymatic activity to cleave a separate ribonucleic acid molecule, comprising:
- a nucleotide base-containing molecule having a hammerhead motif which has the formula 3′-V-W-X-Y-Z-5′, wherein V and Z are sequences which can base pair with said separate ribonucleic acid molecule and X is a sequence which can base pair inter se to form a hairpin;
- and wherein W comprises 3′-AbAAG-5, Y comprises two 3′-AGb-5′;
- wherein b represents a nucleotide having a 2′-OH group;
- and wherein at least one of the sugar moieties in said enzymatic nucleic acid molecule comprises, at its 2′-position, an analogue group other than hydroxyl alone, wherein said analogue group is not 2′-alkoxy or 2′-halo, and does not comprise a nitrogen atom linked to the 2′-C, wherein said analogue group at each 2′-position may be the same or different;
- and a pharmaceutically acceptable carrier.
3 more elements
A pharmaceutical composition, comprising:
- at least one enzymatic nucleic acid molecule having a ribonucleotide at a catalytically critical site and at least one deoxyribonucleotide or nucleic acid analog, and has catalytic activity to cleave a separate ribonucleic acid molecule, wherein said enzymatic nucleic acid molecule comprises
- a nucleotide base-containing molecule having a hairpin motif;
- and a pharmaceutically acceptable carrier.
Dependencies are inferred from the claim text.