CN114787358B - 细胞的细胞类型选择性免疫保护 - Google Patents

细胞的细胞类型选择性免疫保护

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Publication number
CN114787358B
CN114787358B CN202080065723.3A CN202080065723A CN114787358B CN 114787358 B CN114787358 B CN 114787358B CN 202080065723 A CN202080065723 A CN 202080065723A CN 114787358 B CN114787358 B CN 114787358B
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cells
hla
expression
gene
cell
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CN114787358A (zh
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S·A·戈德曼
A·本拉斯
C·特罗杰尔-汉森
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University of Rochester
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University of Rochester
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    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/70539—MHC-molecules, e.g. HLA-molecules
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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    • C12N2310/00—Structure or type of the nucleic acid
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CN202080065723.3A 2019-07-18 2020-07-20 细胞的细胞类型选择性免疫保护 Active CN114787358B (zh)

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Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014124087A1 (en) 2013-02-06 2014-08-14 University Of Rochester Induced pluripotent cell-derived oligodendrocyte progenitor cells for the treatment of myelin disorders
US9724432B2 (en) 2015-04-30 2017-08-08 University Of Rochester Non-human mammal model of human degenerative disorder, uses thereof, and method of treating human degenerative disorder
WO2023150553A1 (en) * 2022-02-01 2023-08-10 University Of Rochester Gpr17 promoter-based targeting and transduction of glial progenitor cells
JP7457505B2 (ja) 2017-05-10 2024-03-28 ユニバーシティー オブ ロチェスター 神経精神障害を処置する方法
KR20210054502A (ko) 2018-06-21 2021-05-13 유니버시티 오브 로체스터 헌팅턴병을 치료하거나 이의 발병을 저해하는 방법
BR112022019060A2 (pt) 2020-03-25 2022-11-29 Sana Biotechnology Inc Células neurais hipoimunogênicas para o tratamento de distúrbios e condições neurológicas
US11459372B2 (en) 2020-11-30 2022-10-04 Crispr Therapeutics Ag Gene-edited natural killer cells
US11473060B2 (en) 2020-12-30 2022-10-18 Crispr Therapeutics Ag Compositions and methods for differentiating stem cells into NK cells
CN120898000A (zh) * 2023-01-13 2025-11-04 拉维洛克治疗有限责任公司 新的多核苷酸、细胞和方法
WO2025222093A2 (en) * 2024-04-19 2025-10-23 Factor Bioscience Inc. Engineered cells and uses thereof
WO2025250531A1 (en) * 2024-05-28 2025-12-04 Thymmune Therapeutics, Inc. Hypoimmune engineering for universal ipsc-derived cells

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016183041A2 (en) * 2015-05-08 2016-11-17 President And Fellows Of Harvard College Universal donor stem cells and related methods

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080119427A1 (en) 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6812027B2 (en) 1998-03-25 2004-11-02 Cornell Research Foundation, Inc. Discovery, localization, harvest, and propagation of an FGF2 and BDNF-responsive population of neural and neuronal progenitor cells in the adult human forebrain
US6838283B2 (en) 1998-09-29 2005-01-04 Isis Pharmaceuticals Inc. Antisense modulation of survivin expression
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
PT1152009E (pt) 1999-02-12 2005-03-31 Sankyo Co Novos analogos de nucleosidos e oligonucleotidos
US7098192B2 (en) 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
AU2913801A (en) 1999-12-23 2001-07-03 Cornell Research Foundation Inc. A method for isolating and purifying multipotential neural progenitor cells and multipotential neural progenitor cells
US7179796B2 (en) 2000-01-18 2007-02-20 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
WO2001053503A1 (en) 2000-01-18 2001-07-26 Cornell Research Foundation, Inc. Neural progenitor cells from hippocampal tissue and a method for isolating and purifying them
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
US7150989B2 (en) 2001-08-10 2006-12-19 Cornell Research Foundation, Inc. Telomerase immortalized neural progenitor cells
AU2003251286B2 (en) 2002-01-23 2007-08-16 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
WO2003070171A2 (en) 2002-02-15 2003-08-28 Cornell Research Foundation, Inc. Myelination of congenitally dysmyelinated forebrains using oligodendrocyte progenitor cells
ES2280826T5 (es) 2002-08-05 2017-08-03 Silence Therapeutics Gmbh Nuevas formas adicionales de moléculas de ARN de interferencia
US8642332B2 (en) 2003-03-07 2014-02-04 Cornell Research Foundation, Inc. Identification and isolation of multipotential neural progenitor cells from the subcortical white matter of the adult human brain
US8263406B2 (en) 2003-06-11 2012-09-11 Cornell Research Foundation, Inc. Enriched or purified population of motor neurons and its preparation from a population of embryonic stem cells
SG146682A1 (en) 2003-09-18 2008-10-30 Isis Pharmaceuticals Inc Modulation of eif4e expression
WO2005046610A2 (en) 2003-11-10 2005-05-26 Cornell Research Foundation, Inc. Genes differentially expressed by acutely isolated resident progenitor cells of the human white matter
KR101439568B1 (ko) 2006-08-11 2014-09-12 다우 아그로사이언시즈 엘엘씨 아연 손가락 뉴클레아제-매개 상동 재조합
US8012678B2 (en) 2007-07-24 2011-09-06 Wisconsin Alumni Research Foundation Biomarkers for human papilloma virus-associated cancer
KR20100080068A (ko) 2008-12-31 2010-07-08 주식회사 툴젠 신규한 징크 핑거 뉴클레아제 및 이의 용도
EP3156062A1 (en) 2010-05-17 2017-04-19 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
HK1200871A1 (en) 2011-11-16 2015-08-14 Sangamo Therapeutics, Inc. Modified dna-binding proteins and uses thereof
WO2014124087A1 (en) 2013-02-06 2014-08-14 University Of Rochester Induced pluripotent cell-derived oligodendrocyte progenitor cells for the treatment of myelin disorders
US9393257B2 (en) 2013-03-01 2016-07-19 Regents Of The University Of Minnesota TALEN-based gene correction
EP3004339B1 (en) 2013-05-29 2021-07-07 Cellectis New compact scaffold of cas9 in the type ii crispr system
ES3029138T3 (en) 2013-06-05 2025-06-23 Univ Duke Rna-guided gene editing and gene regulation
EP3011011A4 (en) * 2013-06-19 2017-05-31 Sigma-Aldrich Co. LLC Targeted integration
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
EP3151846A4 (en) 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design
ES2865275T3 (es) 2014-08-06 2021-10-15 College Of Medicine Pochon Cha Univ Industry Academic Cooperation Foundation Células inmunocompatibles creadas por edición mediada por nucleasas, de genes que codifican HLA
EP3294896A1 (en) 2015-05-11 2018-03-21 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016183041A2 (en) * 2015-05-08 2016-11-17 President And Fellows Of Harvard College Universal donor stem cells and related methods

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
"Hypoimmunogenic derivatives of induced pluripotent stem cells evade immune rejection in fully immunocompetent allogeneic recipients;OEUSE. T 等;NATURE BIOTECHNOLOGY;252-258 *
Generation of hypoimmunogenic human pluripotent stem cells;HAN, X. 等;PNAS;第116卷(第21期);第1044页左栏 *

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JP7708737B2 (ja) 2025-07-15
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CN114787358A (zh) 2022-07-22
EP3999627A2 (en) 2022-05-25

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