US2005074760A1PendingUtilityA1

Enzymatic nucleic acids containing 5'-and/or 3'-cap structures

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Assignee: RIBOZYME PHARM INCPriority: Apr 16, 1996Filed: May 6, 2003Published: Apr 7, 2005
Est. expiryApr 16, 2016(expired)· nominal 20-yr term from priority
Inventors:Jasenka Matulic-AdamicLeonid BeigelmanAlexander KarpeiskyThale JarvisNassim UsmanAnthony DirenzoFrancine Wincott
C07H 19/06C07H 19/16C12N 15/113C12N 2310/121C07H 21/00
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Claims

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.

Claims

exact text as granted — not AI-modified
1 . A 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-(β-D-erythrofuranosyl) nucleotide; 1,3-diamino-2-propyl phosphate, 3-aminopropyl phosphate; 12-aminododecyl phosphate; hydroxypropyl phosphate; 1,5-anhydrohexitol nucleotide; L-nucleotide; threo-pentofuranosyl nucleotide; acyclic 3′,4′-seco nucleotide; 3,5-dihydroxypentyl nucleotide; and 5′-mercapto moieties, and wherein said 3′-cap structure is selected from the group consisting of 1,5-anhydrohexitol nucleotide; L-nucleotide; threo-pentofuranosyl nucleotide; acyclic 3′,4′-seco nucleotide; 3,5-dihydroxypentyl nucleotide; 3′-2′-inverted nucleotide moiety; 3′-2′-inverted abasic moiety; and 1,4-butanediol, wherein said nucleic acid molecule is single stranded.  
     
     
         2 . The nucleic acid molecule of  claim 1 , wherein said nucleic acid molecule is in an enzymatic nucleic acid molecule.  
     
     
         3 . The nucleic acid molecule of  claim 1 , wherein said nucleic acid molecule is in an antisense nucleic acid molecule.  
     
     
         4 . The nucleic acid molecule of  claim 2 , wherein said enzymatic nucleic acid molecule is in a hairpin, hepatitis delta virus, group I intron, VS RNA or Rnase P RNA motif.  
     
     
         5 . The nucleic acid molecule of  claim 1 , wherein said nucleic acid molecule comprises said 5′-cap structure.  
     
     
         6 . The nucleic acid molecule of  claim 1 , wherein said nucleic acid molecule comprises said 3′-cap structure.  
     
     
         7 . The nucleic acid molecule of  claim 1 , wherein said nucleic acid molecule comprises said 5′-cap structure and said 3′-cap structure.  
     
     
         8 . The nucleic acid molecule of  claim 1 , wherein said 5′- and 3′-cap structures are different.  
     
     
         9 . The nucleic acid molecule of  claim 1 , wherein said 5′- and 3′-cap structures are same.  
     
     
         10 . The nucleic acid of  claim 1 , wherein said 3′-cap structure is a 3′-2′ linked inverted nucleotide.  
     
     
         11 . The nucleic acid of  claim 1 , wherein said 3′-cap structure is a 3′-32 linked inverted abasic moiety.  
     
     
         12 . The nucleic acid of  claim 1 , wherein said 5′-cap structure is a 1,3-diamino-2-propyl phosphate group.  
     
     
         13 . The nucleic acid of  claim 1 , wherein said 5′-cap structure is a L-nucleotide.  
     
     
         14 . The nucleic acid of  claim 1 , wherein said 5′-cap structure is a threo-pentafuranosyl group.  
     
     
         15 . The nucleic acid of  claim 1 , wherein said 5′-cap structure is a 3,5-dihydroxypentyl nucleotide.  
     
     
         16 . The nucleic acid of  claim 1 , wherein said 5′-cap structure is a 1-(β-D-erythrofuranosyl) nucleotide.  
     
     
         17 . The nucleic acid of  claim 1 , wherein said 3′-cap structure is a L-nucleotide.  
     
     
         18 . The nucleic acid of  claim 1 , wherein said 3′-cap structure is a 3,5-dihydroxypentyl nucletide.  
     
     
         19 . An isolated mammalian cell comprising the nucleic acid molecule of  claim 1 .  
     
     
         20 . The mammalian cell of  claim 19 , wherein said mammalian cell is a human cell.

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