COMPOUNDS AND METHODS FOR MODULATING PROTEIN EXPRESSION
United States Patent Application 20110124591
Kind Code A1
Bennett; C. Frank May 26, 2011
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COMPOUNDS AND METHODS FOR MODULATING PROTEIN EXPRESSION
Abstract
The present invention provides compounds and methods for modulating expression of a protein, including, but not limited to, modulating splicing of a pre-mRNA to modulate the amount of one or more variants of a protein.
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Inventors: Bennett; C. Frank; (Carlsbad, CA)
Assignee: ISIS Pharmaceuticals Inc.
Carlsbad
CA
Serial No.: 742652
Series Code: 12
Filed: November 13, 2008
PCT Filed: November 13, 2008
PCT NO: PCT/US08/83448
371 Date: October 8, 2010
Current U.S. Class: 514/43; 435/455
Class at Publication: 514/43; 435/455
International Class: A61K 31/7088 20060101 A61K031/7088; C12N 15/85 20060101 C12N015/85
BACKGROUND OF THE INVENTION
[0003] Antisense compounds have been used to modulate target nucleic acids. Antisense compounds comprising a variety of modifications and motifs have been reported. In certain instances, such compounds are useful as research tools and as therapeutic agents. In certain instances antisense compounds have been shown to modulate protein expression by altering splicing of a pre-mRNA, by arresting translation and/or by interrupting poly-adenylation of a pre-mRNA.
SUMMARY OF THE INVENTION
[0004] In certain embodiments, provided herein are compounds and methods that modulate protein expression, including, but not limited to, modulation of splicing. |
