Title:
GENETICALLY MODIFIED STEM CELL HAVING INHIBITED B2M GENE EXPRESSION, AND METHOD FOR USING SAME
Document Type and Number:
WIPO Patent Application WO/2023/008918
Kind Code:
A1
Abstract:
The present invention relates to a genetically modified stem cell having reduced expression or activity of the B2M gene or B2M protein, and a method for using same. One aspect relates to pluripotent stem cells (PCNT-PSCs) prepared by preparing somatic cells, of which the B2M gene has been knocked out, and subjecting same to somatic cell nuclear transfer, and immune cells induced to differentiate from same. The pluripotent stem cells and immune cells of the aspect are capable of differentiation into NK cells and gamma delta T cells, which are normal immune cells, even if the B2M gene has been knocked out. Further, since HLA class I molecules are induced to be knocked out by targeting the B2M gene, the prepared cells can survive in the body for a long time and minimize the number of injections through "off-the-shelf" immune rejection escape which does not give rise to a reaction of allogeneic T cells, and thus may be utilized as low-immune cells or immune rejection escape stem cells and immune cells and thus widely utilized as a cell therapy product.
Inventors:
LEE AH REUM (KR)
LEE JI YOON (KR)
LEE JI YOON (KR)
Application Number:
PCT/KR2022/011100
Publication Date:
February 02, 2023
Filing Date:
July 28, 2022
Export Citation:
Assignee:
SUNGKWANG MEDICAL FOUND (KR)
International Classes:
C12N5/0783; A61K48/00; C12N5/0735; C12N9/22; C12N15/10; C12N15/113
Foreign References:
| KR20160139045A | 2016-12-06 | |||
| KR20210032449A | 2021-03-24 | |||
| KR20190137010A | 2019-12-10 |
Other References:
BOGOMIAKOVA M.E., SEKRETOVA E.K., EREMEEV A.V., SHUVALOVA L.D., BOBROVSKY P.A., ZERKALENKOVA E.A., LEBEDEVA O.S., LAGARKOVA M.A.: "Derivation of induced pluripotent stem cells line (RCPCMi007-A-1) with inactivation of the beta-2-microglobulin gene by CRISPR/Cas9 genome editing", STEM CELL RESEARCH, ELSEVIER, NL, vol. 55, 1 August 2021 (2021-08-01), NL , pages 102451, XP093029427, ISSN: 1873-5061, DOI: 10.1016/j.scr.2021.102451
ZHA SHIJUN, TAY JOHAN CHIN-KANG, ZHU SUMIN, LI ZHENDONG, DU ZHICHENG, WANG SHU: "Generation of Mesenchymal Stromal Cells with Low Immunogenicity from Human PBMC-Derived β2 Microglobulin Knockout Induced Pluripotent Stem Cells", CELL TRANSPLANTATION, SAGE, US, vol. 29, 1 January 2020 (2020-01-01), US , pages 096368972096552, XP093029428, ISSN: 0963-6897, DOI: 10.1177/0963689720965529
ZHA SHIJUN, TAY JOHAN CHIN-KANG, ZHU SUMIN, LI ZHENDONG, DU ZHICHENG, WANG SHU: "Generation of Mesenchymal Stromal Cells with Low Immunogenicity from Human PBMC-Derived β2 Microglobulin Knockout Induced Pluripotent Stem Cells", CELL TRANSPLANTATION, SAGE, US, vol. 29, 1 January 2020 (2020-01-01), US , pages 096368972096552, XP093029428, ISSN: 0963-6897, DOI: 10.1177/0963689720965529
Attorney, Agent or Firm:
Y.P.LEE, MOCK & PARTNERS (KR)
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