Title:
遺伝子改変細胞をポジティブに選択して、排除するための短ヘアピンＲＮＡ（ＳＨＲＮＡ７３４）およびその使用
Document Type and Number:
Japanese Patent JP6975465
Kind Code:
B2
Abstract:
A potent short hairpin RNA (shRNA734) directed to human Hypoxanthine Guanine Phosphoribosyltransferase (HPRT) improves the rate of gene-modified stem cell engraftment by a conditioning and in vivo selection strategy to confer resistance to a clinically available guanine analog antimetabolite, 6TG, for efficient positive selection of gene-modified stem cells. Uses for polynucleotides comprising the shRNA734 include methods for knocking down HPRT in a cell, for conferring resistance to a guanine analog antimetabolite in a cell, for producing selectable genetically modified cells, for selecting cells genetically modified with a gene of interest from a plurality of cells, for removing cells genetically modified with a gene of interest from a plurality of cells, and for treating a subject infected with HIV.
Inventors:
Ann, Dong Sun
Shimizu, Saki
Shimizu, Saki
Application Number:
JP2018543719A
Publication Date:
December 08, 2021
Filing Date:
February 17, 2017
Export Citation:
Assignee:
The Regents of the University of California
International Classes:
C12N15/113; A61K31/7088; A61K35/12; A61K35/76; A61P31/18; A61P43/00; C12N5/10
Domestic Patent References:
| JP2010520757A |
Other References:
HACKE, K., et al.,"Genetic modification of mouse bone marrow by lentiviral vector-mediated delivery of HPRT shRNA confers chemoprotection against 6-thioguanine cytotoxicity.",Transplant Proc.,2013年,Vol.45, No.5,pp.2040-2044
NARUKAWA, M., et al.,"Efficient selection of genetically engineered HIV resistant cells by short hairpin RNA mediated HPRT and CCR5 knockdown.",Molecular Therapy,2013年05月,Vol.21, No.suppl.1,S204,[503]
NARUKAWA, M., et al.,"Efficient selection of genetically engineered HIV resistant cells by short hairpin RNA mediated HPRT and CCR5 knockdown.",International Journal of Antimicrobial Agents,2013年,Vol.42S2,p.S128,[P274]
NARUKAWA, M., et al.,"Efficient selection of genetically engineered HIV resistant cells by short hairpin RNA mediated HPRT and CCR5 knockdown.",Molecular Therapy,2013年05月,Vol.21, No.suppl.1,S204,[503]
NARUKAWA, M., et al.,"Efficient selection of genetically engineered HIV resistant cells by short hairpin RNA mediated HPRT and CCR5 knockdown.",International Journal of Antimicrobial Agents,2013年,Vol.42S2,p.S128,[P274]
Attorney, Agent or Firm:
Ryoichi Takaoka
Nao Oda
Akiyo Iwahori
Kaoru Takahashi
Nao Oda
Akiyo Iwahori
Kaoru Takahashi