Title:
CUSTOMIZED CCR5/CXCR4-GENE SIMULTANEOUS KNOCKOUT HEMATOPOIETIC STEM CELLS FOR TREATMENT OR PREVENTION OF HIV INFECTION, AND PREPARATION METHOD THEREFOR
Document Type and Number:
WIPO Patent Application WO/2021/107234
Kind Code:
A1
Abstract:
The present invention relates to a method for preparing customized CCR5/CXCR4-gene simultaneous knockout hematopoietic stem cells by using a CRISPR/Cas9 system, and a pharmaceutical composition and a cell therapeutic agent for treating or preventing HIV infection, comprising the hematopoietic stem cells. The method for preparing a hematopoietic stem cell, of the present invention, can efficiently prepare hematopoietic stem cells in which CCR5 and CXCR4 genes are simultaneously knocked out, and thus hematopoietic stem cells prepared thereby can be used as a pharmaceutical composition and cell therapeutic agent for treating or preventing HIV infection.
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Inventors:
KANG KYUNG SUN (KR)
SEO KWANG WON (KR)
KWON DAEKEE (KR)
HAN MI JUNG (KR)
SEO KWANG WON (KR)
KWON DAEKEE (KR)
HAN MI JUNG (KR)
Application Number:
PCT/KR2019/016796
Publication Date:
June 03, 2021
Filing Date:
November 29, 2019
Export Citation:
Assignee:
KANGSTEM BIOTECH CO LTD (KR)
International Classes:
C12N5/0789; A61K35/28; A61P31/18
Foreign References:
| KR20080075959A | 2008-08-20 | |||
| US20120171771A1 | 2012-07-05 | |||
| KR20190135946A | 2019-12-09 |
Other References:
YU SONGLIN, YAO YONGCHAO, XIAO HONGKUI, LI JIAOJIAO, LIU QUAN, YANG YIJUN, ADAH DICKSON, LU JUNNAN, ZHAO SITING, QIN LI, CHEN XIAO: "Simultaneous knockout of CXCR4 and CCR5 genes in CD 4+ T cells via CRISPR/Cas9 confers resistance to both X4-and R5-tropic human immunodeficiency vims type 1 infection", HUMAN GENE THERAPY, vol. 29, no. 1, 2018, pages 51 - 67, XP055832611
LINDROOS BETTINA; BOUCHER SHAYNE; CHASE LUCAS; KUOKKANEN HANNU; HUHTALA HEINI; HAATAJA RIINA; VEMURI MOHAN; SUURONEN RIITTA; MIETT: "Serum-free, xeno-free culture media maintain the proliferation rate LINDROOS, B. et al. Serum-free, xeno-free culture media maintain the proliferation rate and multipotentiality of adipose stem cells in vitro", CYTOTHERAPY, vol. 11, no. 7, 2009, pages 958 - 972, XP009157099
PANPAN HOU; SHULIANG CHEN; SHILEI WANG; XIAO YU; YU CHEN; MENG JIANG; KE ZHUANG; WENZHE HO; WEI HOU; JIAN HUANG; DEYIN GUO: "Genome editing of CXCR4 by CRISPR/cas9 confers cells resistant to HIV-1 infection", SCIENTIFIC REPORTS, vol. 5, no. 1, 2015, pages 15577, XP055267475
LINDROOS BETTINA; BOUCHER SHAYNE; CHASE LUCAS; KUOKKANEN HANNU; HUHTALA HEINI; HAATAJA RIINA; VEMURI MOHAN; SUURONEN RIITTA; MIETT: "Serum-free, xeno-free culture media maintain the proliferation rate LINDROOS, B. et al. Serum-free, xeno-free culture media maintain the proliferation rate and multipotentiality of adipose stem cells in vitro", CYTOTHERAPY, vol. 11, no. 7, 2009, pages 958 - 972, XP009157099
PANPAN HOU; SHULIANG CHEN; SHILEI WANG; XIAO YU; YU CHEN; MENG JIANG; KE ZHUANG; WENZHE HO; WEI HOU; JIAN HUANG; DEYIN GUO: "Genome editing of CXCR4 by CRISPR/cas9 confers cells resistant to HIV-1 infection", SCIENTIFIC REPORTS, vol. 5, no. 1, 2015, pages 15577, XP055267475
Attorney, Agent or Firm:
SON, Min (KR)
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