IP Library › Granted Patent US 11,434,476
Granted Patent B2
US 11,434,476 · App. 16/326,700 · Granted Sep 6, 2022

Methods of editing DNA methylation

Inventors: Rudolf Jaenisch (Brookline, MA); X Shawn Liu (Cambridge, MA); Hao Wu (Newton, MA)
Assignee: Whitehead Institute for Biomedical Research
C12N9/22A61K35/30A61K35/33C12N9/1007C12N15/11C12N15/86C12N15/907C12Y201/01037C12N2310/20C12N2740/15031C12N2800/80
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Quick Facts
Patent No.
US 11,434,476
App. No.
16/326,700
Granted
Sep 6, 2022
Kind
B2
Abstract

The invention relates to methods of modifying DNA methylation by contacting a catalytically inactive site specific nuclease fused to an effector domain having methylation or demethylation activity and one or more guide sequences.

Claims (24)

1. A method of modulating the methylation of one or more CTCF binding sites in a cell comprising introducing into the cell

a. a catalytically inactive site specific nuclease fused to an effector domain having methylation activity, wherein the effector domain comprises Dnmt3a; and

b. one or more guide sequences or a nucleic acid that encodes the one or more guide sequences, wherein the one or more guide sequences target the CTCF binding sites, thereby modulating the methylation of one or more genomic sequences in the cell.

2. The method of claim 1 , wherein the catalytically inactive site specific nuclease is a catalytically inactive Cas protein,

wherein the catalytically inactive site specific nuclease is a catalytically inactive Cas9 protein, or

wherein the catalytically inactive site specific nuclease is a catalytically inactive Cpf1 protein.

3. The method of claim 1 , wherein the one or more guide sequences are ribonucleic acid guide sequences.

4. The method of claim 1 , wherein the one or more guide sequences are from about 10 base pairs to about 150 base pairs in length.

5. The method of claim 1 , wherein the one or more guide sequences comprises two or more guide sequences.

6. The method of claim 1 , wherein 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, or 20 genomic sequences are modified in the cell.

7. The method of claim 1 , wherein the cell is a stem cell, a neuron, a post-mitotic cell, or a fibroblast.

8. The method of claim 1 , wherein the cell is a human cell.

9. A method of modulating the methylation of one or more enhancers of MyoD in a cell comprising introducing into the cell

a. a catalytically inactive site specific nuclease fused to an effector domain having demethylation activity, wherein the effector domain comprises Tet1; and

b. one or more guide sequences or a nucleic acid that encodes one or more guide sequences, wherein the one or more guide sequences target the enhancers of MyoD, thereby modulating the methylation of one or more genomic sequences in the cell.

10. The method of claim 9 , wherein the catalytically inactive site specific nuclease is a catalytically inactive Cas protein,

wherein the catalytically inactive site specific nuclease is a catalytically inactive Cas9 protein, or

wherein the catalytically inactive site specific nuclease is a catalytically inactive Cpf1 protein.

11. The method of claim 9 , wherein the one or more guide sequences are ribonucleic acid guide sequences.

12. The method of claim 9 , wherein the one or more guide sequences are from about 10 base pairs to about 150 base pairs in length.

13. The method of claim 9 , wherein the one or more guide sequences comprises two or more guide sequences.

14. The method of claim 9 , wherein 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, or 20 genomic sequences are modified in the cell.

15. The method of claim 9 , wherein the cell is a stem cell, a neuron, a post-mitotic cell, or a fibroblast.

16. The method of claim 9 , wherein the cell is a human cell.

Assignments (2)
SECURITY INTEREST Recorded Feb 10, 2025
From: OMEGA THERAPEUTICS, INC.
To: PIONEERING MEDICINES 08- B, INC.
Reel/Frame 070167/0243 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Apr 12, 2021
From: JAENISCH, RUDOLF; LIU, X SHAWN; WU, HAO
To: WHITEHEAD INSTITUTE FOR BIOMEDICAL RESEARCH
Reel/Frame 055890/0920 →
Continuity (2)
Provisional Application 62377520 · Aug 19, 2016
Related Publication 20190359959A1 · Nov 28, 2019
Cited By (4)
US 12,234,453 US 12,509,683 US 12,522,823 US 12,553,037