Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
AssigneeRibozyme Pharmaceuticals, Inc.
InventorsJasenka Matulic-Adamic, Leonid Beigelman, Alexander Karpeisky, Thale Jarvis, Nassim Usman, Anthony DiRenzo, Francine Wincott
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Abstract
An enzymatic nucleic acid molecule comprising a 5'- and/or a 3'-cap structure, wherein said structure is not a 5'-5'-linked inverted nucleotide or a 3'-3'-linked inverted nucleotide.
Claims41
An enzymatic nucleic acid molecule comprising a 5'-cap structure, a 3'-cap structure, or both a 5'- and a 3'-cap structure, wherein said 5'-cap structure is selected from the group consisting of 4',5'-methylene nucleotide;
- 1-(.beta.-D-erythrofuranosyl)nucleotide;
- 4'-thio nucleotide, carbocyclic nucleotide;
- 5'-amino-alkyl phosphate;
- 1,3-diamino-2-propyl phosphate, 3-aminopropyl phosphate;
- 6-aminohexyl phosphate;
- 12-aminododecyl phosphate;
- hydroxypropyl phosphate;
- 1,5-anhydrohexitol nucleotide;
- L-nucleotide;
- alpha-nucleotide;
- threo-pentofuranosyl nucleotide;
- acyclic 3',4'-seco nucleotide;
- 3,4-dihydroxybutyl nucleotide;
- 3,5-dihydroxypentyl nucleotide, 5'-5'-inverted abasic moiety;
- 1,4-butanediol phosphate;
- 5'-amino;
- and bridging or non bridging methylphosphonate and 5'-mercapto moieties, and wherein said 3'-cap structure is selected from the group consisting of 4'-thio nucleoside, carbocyclic nucleotide;
- 1,5-anhydrohexitol nucleotide;
- L-nucleotide;
- alpha-nucleotide;
- modified base nucleotide;
- threo-pentofuranosyl nucleotide;
- acyclic 3',4'-seco nucleotide;
- 3,4-dihydroxybutyl nucleotide;
- 3,5-dihydroxypentyl nucleotide;
- 3'-3'-inverted abasic moiety;
- 3'-2'-inverted nucleotide moiety;
- 3'-2'-inverted abasic moiety;
- 1,4-butanediol;
- hexylphosphate;
- aminohexyl phosphate;
- 3'-phosphate;
- and a bridging methylphosphonate moiety.
A method for producing an enzymatic nucleic acid molecule having activity to cleave an RNA or a single-stranded DNA molecule, comprising the step of forming said enzymatic nucleic acid molecule with a 5'-cap structure, a 3'-cap structure, or both a 5'- and a 3'-cap structure,
- wherein said 5'-cap structure is selected from the group consisting of 4',4'-methylene nucleotide;
- 1-(.beta.-D-erythrofuranosyl)nucleotide;
- 4'-thio nucleotide, carbocyclic nucleotide;
- 5'-amino-alkyl phosphate;
- 1,3-diamino-2-propyl phosphate, 3-aminopropyl phosphate;
- 6-aminohexyl phosphate;
- 12-aminododecyl phosphate;
- hydroxypropyl phosphate;
- 1,5-anhydrohexitol nucleotide;
- L-nucleotide;
- alpha-nucleotide;
- threo-pentofuranosyl nucleotide;
- acyclic 3',4'-seco nucleotide;
- 3,4-dihydroxybutyl nucleotide;
- 3,5-dihydroxypentyl nucleotide, 5'-5'-inverted abasic moiety;
- 1,4-butanediol phosphate;
- 5'-amino;
- and bridging or non bridging methylphosphonate and 5'-mercapto moieties, and wherein said 3'-cap structure is selected from the group consisting of 4'-thio nucleoside, carbocyclic nucleotide;
- 1,5-anhydrohexitol nucleotide;
- L-nucleotide;
- alpha-nucleotide;
- modified base nucleotide;
- threo-pentofuranosyl nucleotide;
- acyclic 3',4'-seco nucleotide;
- 3,4-dihydroxybutyl nucleotide;
- 3,5-dihydroxypentyl nucleotide;
- 3'-3'-inverted abasic moiety;
- 3'-2'-inverted nucleotide moiety;
- 3'-2'-inverted abasic moiety;
- 1,4-butanediol;
- hexylphosphate;
- aminohexyl phosphate;
- 3'-phosphate;
- and a bridging methylphosphonate moiety.
Dependencies are inferred from the claim text.