IP Library Granted Patent US 9,518,261
Granted Patent B2
US 9,518,261 · App. 14/403,103 · Granted Dec 13, 2016

Modulation of enhancer RNA mediated gene expression

Inventors: Susan M. Freier (San Diego, CA); Christopher K. Glass (San Diego, CA); Michael G. Rosenfeld (San Diego, CA); Wenbo Li (San Diego, CA); Michael T. Lam (La Jolla, CA)
Assignees: Ionis Pharmaceuticals, Inc.; The Regents of the University of California
C12N15/113C12N15/85C12N2310/11C12N2310/113C12N2310/315C12N2310/3231C12N2310/3341C12N2310/341
View Patent ↗
Loading inventors, assignments & file history…
Monitor This Case
Get email alerts when status or documents change.
Order Certified Copies
Most orders are placed with the USPTO same day — all within 24 business hours.
Order via The Patent Place →
Pre-filled with this patent's details
Quick Facts
Patent No.
US 9,518,261
App. No.
14/403,103
Granted
Dec 13, 2016
Kind
B2
Abstract

Disclosed herein are methods and compounds for inhibiting gene expression by inhibiting enhancer RNAs (eRNAs). Such methods and compounds are useful for reducing expression of certain genes, many of which are associated with a variety of diseases and disorders.

Claims (18)

1. A method of inhibiting MMP9 gene expression in a mammalian cell comprising contacting the mammalian cell with a single-stranded antisense compound consisting of the sequence of SEQ ID NO: 3, wherein the antisense compound comprises:

a gap segment consisting of linked deoxynucleosides;

a 5′ wing segment consisting of linked nucleosides; and

a 3′ wing segment consisting of linked nucleosides;

wherein the gap segment is positioned between the 5′ wing segment and the 3′ wing segment, wherein each nucleoside of each wing segment comprises a modified sugar, wherein the antisense compound targets an enhancer RNA (eRNA) transcribed from a genomic enhancer sequence or region, wherein the eRNA is an MMP9 eRNA sequence comprising the nucleic acid sequence of SEQ ID NO: 1, thereby inhibiting expression of the MMP9 gene in the mammalian cell.

2. The method of claim 1 , wherein the eRNA transcription is initiated from a RNA polymerase II (PolII) binding site and is capable of elongating bidirectionally.

3. The method of claim 2 , wherein the eRNA is capable of enhancing transcription of the MMP9 gene.

4. The method of claim 3 , wherein the genomic enhancer sequence or region has a higher level of monomethylated lysine 4 of histone 3 (H3K4me1) than trimethylated lysine 4 of histone 3 (H3K4me3).

5. The method of claim 3 , wherein the genomic enhancer sequence or region is enriched for bound RNA polymerase II (PolII).

6. The method of claim 3 , wherein the genomic enhancer sequence or region is enriched for bound Rev-Erbα or Rev-Erbβ.

7. The method of claim 1 , wherein the transcriptional start site of the MMP9 gene is located on a chromosome at least about 1 kilobase (kb) from the genomic enhancer sequence or region.

8. The method of claim 1 , wherein the mammalian cell is a hematopoietic cell, a monocyte, a macrophage, a neuron, a breast cell, or a cancer cell.

9. The method of claim 8 , wherein the mammalian cell contacted with the antisense compound is in a subject.

10. A compound comprising a single-stranded antisense compound consisting of the sequence of SEQ ID NO: 3, wherein the antisense compound comprises:

a gap segment consisting of linked deoxynucleosides;

a 5′ wing segment consisting of linked nucleosides; and

a 3′ wing segment consisting of linked nucleosides;

wherein the gap segment is positioned between the 5′ wing segment and the 3′ wing segment, wherein each nucleoside of each wing segment comprises a modified sugar, targeting an enhancer RNA (eRNA) transcribed from a genomic enhancer sequence or region, wherein the eRNA is an MMP9 eRNA sequence comprising the nucleic acid sequence of SEQ ID NO:1.

Assignments (2)
CONFIRMATORY LICENSE Recorded Apr 11, 2017
From: UNIVERSITY OF CALIFORNIA SAN DIEGO
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 042214/0735 →
CHANGE OF NAME Recorded May 17, 2016
From: ISIS PHARMACEUTICALS, INC.
To: IONIS PHARMACEUTICALS, INC.
Reel/Frame 038726/0708 →
Continuity (2)
Provisional Application 61650426 · May 22, 2012
Related Publication 20150176005A1 · Jun 25, 2015