Compositions and methods for modulation of lmna expression
AssigneeIsis Pharmaceuticals, Inc.
InventorsC. Frank Bennett, Kenneth W. Dobie, Susan M. Freier, Stanley T. Crooke
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Abstract
Disclosed herein are compounds, compositions and methods for modulating the expression of LMNA in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Further provided are methods of identifying cis splicing regulatory elements of a selected mRNA using the disclosed compounds.
Claims37
A method of identifying a cis splicing regulatory element of a selected pre-mRNA, comprising:
- selecting a splice site of said pre-mRNA;
- designing a set of antisense compounds targeting a region up to about 500 nucleotides 5′ or 3′ of the selected splice site, wherein said antisense compounds are RNase H-independent compounds;
- and
- screening said antisense compounds to identify compounds that modulate the ratio of splicing products of said pre-mRNA.
3-4. (canceled)
6-7. (canceled)
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17-19. (canceled)
21-24. (canceled)
An antisense oligonucleotide 12 to 30 nucleobases in length targeted to and specifically hybridizable with a nucleic acid molecule encoding human LMNA (SEQ ID NO:
- 4), wherein said antisense oligonucleotide comprises at least one 2′-O-(2-methoxyethyl) nucleotide.
26-27. (canceled)
The antisense oligonucleotide of claim 27, comprising a first region comprising one or more deoxynucleotides and second and third regions flanking said first region, each comprising at least one 2′-O-(2-methoxyethyl) nucleotide.
(canceled)
31-32. (canceled)
An antisense oligonucleotide 12 to 30 nucleobases in length targeted to and specifically hybridizable with a nucleic acid molecule encoding human LMNA (SEQ ID NO:
- 4), wherein said antisense oligonucleotide comprises a modified sugar moiety at each nucleotide.
36-39. (canceled)
(canceled)
(canceled)
49-53. (canceled)
Dependencies are inferred from the claim text.