IP Library › Granted Patent US 12,497,601
Granted Patent B2
US 12,497,601 · App. 18/130,202 · Granted Dec 16, 2025

RNA-guided endonuclease fusion polypeptides and methods of use thereof

Inventor: Schaked Omer Halperin (Berkeley, CA)
Assignee: The Regents of the University of California
C12N9/22C12N9/1252C12N9/78C12N15/1024C12N15/11C12Y207/07007C12Y305/04004C12Y305/04005C12N2800/80
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Quick Facts
Patent No.
US 12,497,601
App. No.
18/130,202
Granted
Dec 16, 2025
Kind
B2
Abstract

The present disclosure provides a fusion polypeptide comprising: a) an enzymatically active RNA-guided endonuclease that introduces a single-stranded break in a target DNA; and b) an error-prone DNA polymerase. The present disclosure provides a system comprising: a) a fusion polypeptide of the present disclosure; and b) a guide RNA. The present disclosure provides a cell comprising a fusion polypeptide of the present disclosure, or a system of the present disclosure. The present disclosure provides a method of mutagenizing a target polynucleotide.

Claims (56)

1 . An editing system comprising:

a) an enzymatically active RNA-guided endonuclease that introduces a break in at least one strand in a target DNA;

b) a DNA polymerase that binds to the target DNA 5′ to the break; and

c) a guide RNA that binds the endonuclease,

wherein the endonuclease, DNA polymerase, and guide RNA are exogenously introduced into a cell, or are encoded by one or more exogenously introduced nucleic acids, wherein the endonuclease and the DNA polymerase are fused as parts of a fusion polypeptide, and

wherein the fusion polypeptide does not comprise a deaminase.

2 . The system of claim 1 , wherein the guide RNA comprises a protein-binding segment comprising a nucleotide sequence that binds to the endonuclease, and a target-binding segment comprising a nucleotide sequence that is complementary to a target nucleotide sequence of the target DNA.

3 . The system of claim 1 , wherein the guide RNA comprises one or more of:

a) a modified base;

b) a modified backbone;

c) a modified sugar moiety;

d) a non-natural internucleoside linkage.

4 . The system of claim 1 , wherein the guide RNA guides the endonuclease to introduce the break in the target DNA.

5 . The system of claim 1 , wherein the break in at least one strand is a single-stranded break.

6 . The system of claim 1 , wherein the DNA polymerase synthesizes a new strand on the target DNA.

7 . The system of claim 6 , wherein the DNA polymerase introduces a mutation in the new strand.

8 . The system of claim 7 , wherein the DNA polymerase introduces a mutation in the new strand at a distance of from 1 nucleotide to 100 nucleotides from the single-stranded break in the target DNA.

9 . The system of claim 1 , wherein the endonuclease comprises a class 2 CRISPR/Cas endonuclease.

10 . The system of claim 9 , wherein the class 2 CRISPR/Cas endonuclease comprises a type V or type VI CRISPR/Cas endonuclease.

11 . The system of claim 9 , wherein the class 2 CRISPR/Cas endonuclease comprises a Cas9 polypeptide.

12 . The system of claim 9 , wherein the class 2 CRISPR/Cas endonuclease comprises a Streptococcus pyogenes Cas9 polypeptide.

13 . The system of claim 1 , wherein the fusion polypeptide, when complexed with a guide RNA, exhibits a target mutation rate of from 10 −8 to 10 −7 , 10 −7 to 10 −6 , 10 −6 to 10 −5 , 10 −5 to 10 −4 , 10 −4 to 10 −3 , or 10 −3 to 10 −2 mutations per nucleotide per genome replication event.

14 . The system of claim 1 , wherein the fusion polypeptide further comprises a linker connecting the endonuclease and the DNA polymerase.

15 . The system of claim 1 , wherein the fusion polypeptide further comprises a nuclear localization signal.

16 . The system of claim 1 , wherein the fusion polypeptide comprises, in order from N-terminus to C-terminus:

a) the enzymatically active RNA-guided endonuclease; and

b) the DNA polymerase.

17 . The system of claim 1 , wherein the fusion polypeptide comprises, in order from N-terminus to C-terminus:

a) the enzymatically active RNA-guided endonuclease;

b) a linker; and

c) the DNA polymerase.

18 . The system of claim 1 , wherein the fusion polypeptide comprises, in order from N-terminus to C-terminus:

a) a nuclear localization signal;

b) the enzymatically active RNA-guided endonuclease; and

c) the DNA polymerase.

19 . The system of claim 1 , wherein the fusion polypeptide comprises, in order from N-terminus to C-terminus:

a) a nuclear localization signal;

b) the enzymatically active RNA-guided endonuclease;

c) a linker; and

d) the DNA polymerase.

20 . The system of claim 1 , wherein the fusion polypeptide comprises, in order from N-terminus to C-terminus:

a) the DNA polymerase; and

b) the enzymatically active RNA-guided endonuclease.

21 . The system of claim 1 , wherein the fusion polypeptide comprises, in order from N-terminus to C-terminus:

a) the DNA polymerase;

b) a linker; and

c) the enzymatically active RNA-guided endonuclease.

22 . The system of claim 1 , wherein the fusion polypeptide comprises, in order from N-terminus to C-terminus:

a) a nuclear localization signal;

b) the DNA polymerase; and

c) the enzymatically active RNA-guided endonuclease.

23 . The system of claim 1 , wherein the fusion polypeptide comprises, in order from N-terminus to C-terminus:

a) a nuclear localization signal;

b) the DNA polymerase;

c) a linker; and

d) the enzymatically active RNA-guided endonuclease.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Oct 4, 2023
From: HALPERIN, SCHAKED OMER
To: THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
Reel/Frame 065127/0158 →
Continuity (5)
Division 17340822 · Jun 7, 2021
Continuation 16641950
Provisional Application 62662043 · Apr 24, 2018
Provisional Application 62556127 · Sep 8, 2017
Related Publication 20230399631A1 · Dec 14, 2023
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