IP Library Patent Application 11502158
Patent Application
App. No. 11/502,158

Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof

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Patent No.
US None
App. No.
11/502,158
Abstract

This invention relates generally to chemically modified oligonuceotides useful for modulating expression of microRNAs and pre-microRNAs. More particularly, the invention relates to single stranded chemically modified oligonuceotides for inhibiting microRNA and pre-microRNA expression and to methods of making and using the modified oligonucleotides.

Claims (22)

1 . A method of reducing the amount of a microRNA in a cell in a subject comprising the step of administering an antagomir to the subject, wherein the oligonucletide agent is substantially single-stranded, comprises a sequence which is substantially complementary to 12 to 23 contiguous nucleotides of a target sequence, and wherein the target sequence differs by no more than 1, 2, or 3 nucleotides from a sequence selected from the group consisting of those provided in Table 1.

2 . The method of claim 1 , wherein said antagomir is selected from the group consisting of those provided in Tables 2a-e and Table 4.

3 . An isolated oligonucleotide agent, comprising a nucleotide sequence sufficiently complementary to a microRNA target sequence of about 12 to 23 nucleotides, wherein the target sequence differs by no more than 1, 2, or 3 nucleotides from a sequence selected from the group consisting of those provided in Table 1.

4 . The antagomir of claim 3 , comprising a nucleotide sequence sufficiently complementary to a target sequence of about 15 to 23 nucleotides of the microRNA nucleotide sequence, and wherein the target sequence differs by no more than 1, 2, or 3 nucleotides from a sequence selected from the group consisting of those provided in Table 1.

5 . The antagomir of claim 3 , wherein the antagomir further comprises a non-nucleotide moiety.

6 . The antagomir of claim 3 , wherein the antagomir is stabilized against nucleolytic degradation.

7 . The antagomir of claim 3 , further comprising a phosphorothioate at the first internucleotide linkage at the 5′ end of the nucleotide sequence.

8 . The antagomir of claim 3 , further comprising a phosphorothioate at the first internucleotide linkage at the 3′ end of the nucleotide sequence.

9 . The antagomir of claim 3 , further comprising a phosphorothioate at the first internucleotide linkage at the 5′ end of the nucleotide sequence, and a phosphorothioate at the first internucleotide linkage at the 3′ end of the nucleotide sequence.

10 . The antagomir of claim 3 , further comprising a 2′-modified nucleotide.

11 . The antagomir of claim 10 , wherein the 2′-modified nucleotide comprises a modification selected from the group consisting of: 2′-deoxy, 2′-deoxy-2′-fluoro, 2′-O-methyl, 2′-O-methoxyethyl (2′-O-MOE), 2′-O-aminopropyl (2′-O-AP), 2′-O-dimethylaminoethyl (2′-O-DMAOE), 2′-O-dimethylaminopropyl (2′-O-DMAP), 2′-O-dimethylaminoethyloxyethyl (2′-O-DMAEOE), and 2′-O—N-methylacetamido (2′-O—NMA).

12 . The antagomir of claim 10 , wherein the 2′-modified nucleotide comprises a 2′-O-methyl.

13 . The antagomir of claim 3 , further comprising a cholesterol molecule attached to the 3′ end of the agent.

14 . The antagomir of claim 3 , wherein the antagomir is selected from the group consisting of those provided in Table 2a-e and Table 4.

15 . A method of reducing the amount of miR-122 in a cell, comprising contacting the cell with an antagomir of claim 3 , wherein the sequence selected from the group consisting of those provided in Table 1 is miR-122.

16 . A method of making an antagomir of claim 3 , the method comprising the synthesis of the oligonucleotide agent, wherein the nucleotide sequence comprises at least one modification that stabilizes the antagomir against nucleolytic degradation.

17 . A pharmaceutical composition comprising an antagomir of claim 3 and a pharmaceutically acceptable carrier.

18 . A method of inhibiting miR-122 expression in a cell, comprising contacting the cell with an effective amount of an antagomir of claim 3 , wherein the sequence selected from the group consisting of those provided in Table 1 is miR-122.

19 . A method of increasing aldolase-A protein levels in a cell comprising contacting the cell with an effective amount of an antagomir of claim 3 , wherein the sequence selected from the group consisting of those provided in Table 1 is miR-122.

20 . A method of inhibiting miR-16 expression in a cell, comprising contacting the cell with an effective amount of an antagomir of claim 3 , wherein the sequence selected from the group consisting of those provided in Table 1 is miR-16.

21 . A method of inhibiting miR-192 expression in a cell, comprising contacting the cell with an effective amount of an antagomir of claim 3 , wherein the sequence selected from the group consisting of those provided in Table 1 is miR-192.

22 . A method of inhibiting miR-194 expression in a cell, comprising contacting the cell with an effective amount of an antagomir of claim 3 , wherein the sequence selected from the group consisting of those provided in Table 1 is miR-194.

Assignments (4)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Dec 9, 2014
From: ALNYLAM PHARMACEUTICALS, INC.
To: REGULUS THERAPEUTICS INC.
Reel/Frame 034437/0620 →
CONFIRMATORY LICENSE Recorded Aug 7, 2008
From: ROCKEFELLER UNIVERSITY
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 021353/0841 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 17, 2006
From: STOFFEL, MARKUS
To: THE ROCKEFELLER UNIVERSITY
Reel/Frame 018400/0216 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 17, 2006
From: STOFFEL, MARKUS
To: ROCKEFELLER UNIVERSITY, THE
Reel/Frame 018431/0640 →