Title:
METHOD FOR PREPARING STEM CELL-DERIVED CARDIOMYOPATHY MODEL CELL LINE BY USING CRISPR-BASED BASE EDITING TECHNOLOGY, AND CARDIOMYOPATHY CELL LINE PREPARED BY SAME METHOD
Document Type and Number:
WIPO Patent Application WO/2023/080755
Kind Code:
A1
Abstract:
The present invention relates to a method for preparing a cardiomyopathy model cell line on the basis of 4th generation base editing technology, and the like. The present invention can provide a cell line of an accurate genetic hypertrophic cardiomyopathy model by accurately inducing mutation of only 1 bp base, sgRNA of the present invention is applicable to various human-derived cells, and a cardiomyopathy model cell line prepared by means of generation in an intron that does not have off-target occurrence or does not affect cell phenotype can be used for research on cardiomyopathy, and a therapeutic agent and patient-specific treatment thereof.
Inventors:
LIM DO-SUN (KR)
KIM KYOUNGMI (KR)
NOH JI-MIN (KR)
JEONG TAE YEONG (KR)
JU SUNGJIN (KR)
KIM KYOUNGMI (KR)
NOH JI-MIN (KR)
JEONG TAE YEONG (KR)
JU SUNGJIN (KR)
Application Number:
PCT/KR2022/017408
Publication Date:
May 11, 2023
Filing Date:
November 08, 2022
Export Citation:
Assignee:
UNIV KOREA RES & BUS FOUND (KR)
International Classes:
C12N5/0735; C12N9/22; C12N15/10; C12N15/113
Domestic Patent References:
| WO2021041716A2 | 2021-03-04 |
Foreign References:
| CN108410911A | 2018-08-17 | |||
| CN112522260A | 2021-03-19 |
Other References:
LU JI-ZHEN; QIAO ZHI-BIN; ZHANG LU; CAO HONG-XIA; BAI ZHI-HUI; QI YI-YAO; ZHU HAN-YU; CHEN YA-QI; ZHANG SHOU-MEI; YAN XIU-HUA; BAO: "Generation of a laminopathies-specific iPSC line EHTJUi005-A-3 with homozygous knockout of the LMNA gene by CRISPR/Cas9 technology", STEM CELL RESEARCH, ELSEVIER, NL, vol. 56, 3 September 2021 (2021-09-03), NL , XP086841970, ISSN: 1873-5061, DOI: 10.1016/j.scr.2021.102530
MOSQUEIRA DIOGO, MANNHARDT INGRA, BHAGWAN JAMIE R, LIS-SLIMAK KATARZYNA, KATILI PUSPITA, SCOTT ELIZABETH, HASSAN MUSTAFA, PRONDZYN: "CRISPR/Cas9 editing in human pluripotent stem cell-cardiomyocytes highlights arrhythmias, hypocontractility, and energy depletion as potential therapeutic targets for hypertrophic cardiomyopathy", EUROPEAN HEART JOURNAL, OXFORD UNIVERSITY PRESS, GB, vol. 39, no. 43, 14 November 2018 (2018-11-14), GB , pages 3879 - 3892, XP055852374, ISSN: 0195-668X, DOI: 10.1093/eurheartj/ehy249
MOSQUEIRA DIOGO, MANNHARDT INGRA, BHAGWAN JAMIE R, LIS-SLIMAK KATARZYNA, KATILI PUSPITA, SCOTT ELIZABETH, HASSAN MUSTAFA, PRONDZYN: "CRISPR/Cas9 editing in human pluripotent stem cell-cardiomyocytes highlights arrhythmias, hypocontractility, and energy depletion as potential therapeutic targets for hypertrophic cardiomyopathy", EUROPEAN HEART JOURNAL, OXFORD UNIVERSITY PRESS, GB, vol. 39, no. 43, 14 November 2018 (2018-11-14), GB , pages 3879 - 3892, XP055852374, ISSN: 0195-668X, DOI: 10.1093/eurheartj/ehy249
Attorney, Agent or Firm:
MUHANN PATENT & LAW FIRM (KR)
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