Antisense modulation of nuclear hormone receptors
AssigneeSarepta Therapeutics, Inc.
InventorPatrick L. Iversen
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Abstract
Provided are antisense oligonucleotides and other agents that target and modulate nuclear hormone receptors (NHRs) such as the glucocorticoid receptor (GR), compositions that comprise the same, and methods of use thereof.
Claims16
An antisense oligomer of 22 to 40 bases comprising a targeting sequence selected from any one of SEQ ID NOs:2-14, wherein the antisense oligomer is a morpholino oligomer, and the targeting sequence is complementary to a pre-mRNA transcript of a glucocorticoid receptor (GR), wherein the oligomer alters splicing of the GR pre-mRNA transcript and increases expression of a ligand independent form of the GR.
An antisense oligomer of 10 to 40 bases comprising a targeting sequence of at least 10 contiguous bases complementary to a target sequence, wherein the antisense oligomer is a morpholino oligomer, the target sequence is a pre-mRNA transcript of a glucocorticoid receptor (GR), and the target sequence is set forth in SEQ ID NO:1, wherein the oligomer alters splicing of the GR pre-mRNA transcript and increases expression of a ligand independent form of the GR.
Dependencies are inferred from the claim text.