IP Library › Granted Patent US 12,612,648
Granted Patent B2
US 12,612,648 · App. 17/311,137 · Granted Apr 28, 2026

Production method for genome-edited cells

Inventors: Atsushi Suzuki (Fukuoka, JP); Masaki Kawamata (Fukuoka, JP)
Assignee: KYUSHU UNIVERSITY, NATIONAL UNIVERSITY CORPORATION
C12N15/902C12N9/22C12N15/11G01N21/6486G16B30/00C12N2310/20C12N2800/80
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 12,612,648
App. No.
17/311,137
Granted
Apr 28, 2026
Kind
B2
Abstract

A production method for a cell in which only one allele is genome-edited includes: a step of introducing, into a cell, (A) at least one selected from the group consisting of (a1) a guide RNA in which one or more nucleotide residues are added to a 5′-end of a spacer sequence, (a2) a guide RNA containing a spacer sequence having single-base or multiple-base mismatches with respect to a target sequence, and (a3) an expression vector that can cause the guide RNA of (a1) or (a2) to be expressed, and (B) at least one selected from the group consisting of a Cas protein and an expression vector that can cause the Cas protein to be expressed.

Claims (36)

1 . A guide RNA comprising a spacer sequence,

wherein 15 or more nucleotide residues are added to the 5′-end of the spacer sequence, and

wherein said 15 or more nucleotide residues comprise a polycytosine or a polyguanine including 15 or more nucleotide residues.

2 . The guide RNA according to claim 1 ,

wherein the spacer sequence has single-base or multiple-base mismatches with respect to a target sequence.

3 . An expression vector comprising a sequence encoding the guide RNA according to claim 1 .

4 . The expression vector according to claim 3 , wherein the expression vector further comprises a Cas protein coding sequence and enables a Cas protein to be expressed.

5 . The expression vector according to claim 4 ,

wherein the Cas protein is a Cas9 protein.

6 . A production kit for a cell which is genome-edited, the production kit comprising:

(A) at least one guide RNA according to claim 1 and an expression vector comprising a sequence encoding the guide RNA.

7 . The production kit according to claim 6 further comprising:

(B) at least one selected from the group consisting of a Cas protein and an expression vector for the Cas protein.

8 . The guide RNA according to claim 1 ,

wherein the guide RNA further comprises a repeat sequence, and

wherein the spacer sequence targets a target region, and the repeat sequence forms base pairs with an anti-repeat sequence derived from a tracrRNA to form a complex with a Cas protein.

9 . The guide RNA according to claim 1 ,

wherein the guide RNA has at least one feature selected from the group consisting of:

(a) a suppressed cytotoxicity as compared with a guide RNA in which no nucleotide residues are added to the 5′ end of the spacer sequence;

(b) an increased homology directed repair frequency as compared with a guide RNA in which no nucleotide residues are added to the 5′ end of the spacer sequence; and

(c) an increased monoallelic frequency of genome editing as compared with a guide RNA in which no nucleotide residues are added to the 5′ end of the spacer sequence.

10 . A guide RNA, comprising a spacer sequence and a repeat sequence,

wherein the spacer sequence targets a target region, and the repeat sequence forms base pairs with an anti-repeat sequence derived from a tracrRNA to form a complex with a Cas protein,

wherein 15 or more nucleotide residues are added to a 5′-end of the spacer sequence, and wherein said 15 or more nucleotide residues comprise a polycytosine or a polyguanine of 15 or more nucleotide residues,

wherein the guide RNA suppresses cytotoxicity, increases homology directed repair frequency, or increases mono-allelic genome editing frequency in genome editing compared to a guide RNA which does not have a nucleotide residue added to the 5′ end of the spacer sequence.

11 . An expression vector comprising a sequence encoding the guide RNA according to claim 10 .

12 . The guide RNA according to claim 1 ,

wherein the 15 or more nucleotide residues added to the 5′-end of the spacer sequence are 20 or more nucleotide residues.

13 . The guide RNA according to claim 1 ,

wherein the 15 or more nucleotide residues added to the 5′-end of the spacer sequence comprises a guanine nucleotide at the 5′-end of the 15 or more nucleotide residues.

14 . The guide RNA according to claim 1 ,

wherein the 15 or more nucleotide residues added to the 5′-end of the spacer sequence comprise:

a polycytosine consisting of cytosine residues, wherein the polycytosine is at least 5 residues in length,

nucleotide residues consisting of 17 cytosine residues, an adenosine residue, and 2 cytosine residues, in order from the 5′ end,

nucleotide residues consisting of 22 cytosine residues, an adenosine residue, and 2 cytosine residues, in order from the 5′ end, or

nucleotide residues consisting of 27 cytosine residues, an adenosine residue, and 2 cytosine residues, in order from the 5′ end.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jun 4, 2021
From: SUZUKI, ATSUSHI; KAWAMATA, MASAKI
To: KYUSHU UNIVERSITY, NATIONAL UNIVERSITY CORPORATION
Reel/Frame 056443/0652 →
Priority Claims (1)
JP 2018-232946 · Dec 12, 2018 · national
Continuity (1)
Related Publication 20220025405A1 · Jan 27, 2022
References Cited (27)
US 20150059010A1 · Cigan et al. · 2015 [cited by applicant]
US 20150284727A1 · Kim et al. · 2015 [cited by applicant]
US 20160289675A1 · Ryan · 2016 [cited by examiner]
US 20170355985A1 · Dellinger · 2017 [cited by examiner]
US 20180282722A1 · Jakimo et al. · 2018 [cited by applicant]
CN 104854241A · 2015 [cited by applicant]
CN 104968784A · 2015 [cited by applicant]
CN 106191116A · 2016 [cited by applicant]
JP 2015523856A · 2015 [cited by applicant]
JP 2016500003A · 2016 [cited by applicant]
WO WO2013176772A1 · 2013 [cited by applicant]
WO WO2014093661A2 · 2014 [cited by applicant]
WO WO2018060238A1 · 2018 [cited by applicant]
Okafor, I.C., et al. 2019 Nucleic Acids Research 47(22): 11880-11888, published online Nov. 12, 2019. (Year: 2019). [cited by examiner]
Farboud, B., et al. 2015 Genetics 199: 959-971. (Year: 2015). [cited by examiner]
Anderson et al., “Systematic Analysis of CRISPR-Cas9 Mismatch Tolerance Reveals Low Levels of Off-Target Activity”, Journ. Biotechnology, 211: 56-65, 2015. [cited by applicant]
International Search Report and Written Opinion for PCT International Patent Application No. PCT/JP2019/048781, mailed Mar. 3, 2020. [cited by applicant]
Kawamata et al., “Development of Genome-Editing techniques that Allele-Selective by CRISPR-Cas9 Activity”, Programs and Abstracts of the Annual Meeting of the Molecular Biology Society of Japan, 3P-0595, 2019. [cited by applicant]
Baker et al., “RAC-tagging: Recombineering and Cas9-assisted targeting for protein tagging and conditional analyses”, [cited by applicant]
European Search Report for European Application No. 19895355.6 mailed Jul. 19, 2022, 12 pages. [cited by applicant]
Hwang et al., “Efficient genome editing in zebrafish using a CRISPR-Cas system”, [cited by applicant]
Chinese Office Action for CN 201980091726.1 mailed Jun. 8, 2023, 15 pages. [cited by applicant]
Xie et al., “sgRNA design for the CRISPR/Cas9 system and evaluation of its off-target effects”, [cited by applicant]
Zhou et al., “Development of two chromogenic assays for the target and off-target effects of gene editing nuclease”, Basic Science Collection of Full Text Database of Chinese Excellent Master's Thesis, No. 2, A006-691, … [cited by applicant]
Japanese Office Action for JP Application No. 2024-147493 mailed Mar. 3, 2026, 8 pages. [cited by applicant]
Shechner et al., “CRISPR Display: A modular method for locus-specific targeting of long noncoding RNAs and synthetic RNA devices in vivo”, [cited by applicant]
Tang et al., “Aptazyme-embedded guide RNAs enable ligand-responsive genome editing and transcriptional activation”, [cited by applicant]