CN116234906A - 血型抗原的修饰 - Google Patents

血型抗原的修饰 Download PDF

Info

Publication number
CN116234906A
CN116234906A CN202180022172.7A CN202180022172A CN116234906A CN 116234906 A CN116234906 A CN 116234906A CN 202180022172 A CN202180022172 A CN 202180022172A CN 116234906 A CN116234906 A CN 116234906A
Authority
CN
China
Prior art keywords
cell
gene
seq
guide rna
sequence
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CN202180022172.7A
Other languages
English (en)
Chinese (zh)
Inventor
W·道德勒
A·梅
S·施雷普费尔
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sana Biotechnology Co ltd
Original Assignee
Sana Biotechnology Co ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Sana Biotechnology Co ltd filed Critical Sana Biotechnology Co ltd
Publication of CN116234906A publication Critical patent/CN116234906A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N15/1137—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 enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/48—Reproductive organs
    • A61K35/54—Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
    • A61K35/545—Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0657—Cardiomyocytes; Heart cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01037—Fucosylgalactoside 3-alpha-galactosyltransferase (2.4.1.37)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/0104—Glycoprotein-fucosylgalactoside alpha-N-acetylgalactosaminyltransferase (2.4.1.40)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01069—Galactoside 2-alpha-L-fucosyltransferase (2.4.1.69)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Developmental Biology & Embryology (AREA)
  • Medicinal Chemistry (AREA)
  • Reproductive Health (AREA)
  • Virology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Rheumatology (AREA)
  • Cardiology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CN202180022172.7A 2020-01-13 2021-01-12 血型抗原的修饰 Pending CN116234906A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062960607P 2020-01-13 2020-01-13
US202062960617P 2020-01-13 2020-01-13
US62/960,607 2020-01-13
US62/960,617 2020-01-13
PCT/US2021/013140 WO2021146222A1 (en) 2020-01-13 2021-01-12 Modification of blood type antigens

Publications (1)

Publication Number Publication Date
CN116234906A true CN116234906A (zh) 2023-06-06

Family

ID=74562041

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180022172.7A Pending CN116234906A (zh) 2020-01-13 2021-01-12 血型抗原的修饰

Country Status (7)

Country Link
US (1) US20230272429A1 (https=)
EP (1) EP4090743A1 (https=)
JP (1) JP7853210B2 (https=)
CN (1) CN116234906A (https=)
AU (1) AU2021207810A1 (https=)
CA (1) CA3164591A1 (https=)
WO (1) WO2021146222A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118638907A (zh) * 2024-06-26 2024-09-13 中国人民解放军联勤保障部队第九六〇医院 一种用于检测易漏检类孟买表型的snp标记

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11162079B2 (en) 2019-05-10 2021-11-02 The Regents Of The University Of California Blood type O Rh-hypo-immunogenic pluripotent cells
US12221622B2 (en) 2019-05-10 2025-02-11 The Regents Of The University Of California Modified pluripotent cells
BR112023024231A2 (pt) * 2021-05-19 2024-01-30 Sana Biotechnology Inc Células t primárias rhd negativas hipoimunogênicas
US20240358761A1 (en) 2021-08-11 2024-10-31 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
JP2024535677A (ja) 2021-08-11 2024-10-02 サナ バイオテクノロジー，インコーポレイテッド 即時血液媒介性炎症反応を減少させるための同種細胞療法を目的とした遺伝子改変細胞
EP4384598A1 (en) 2021-08-11 2024-06-19 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions
US20250129346A1 (en) * 2021-08-11 2025-04-24 Sana Biotechnology, Inc. Engineering cells for cell-based therapies, and associated compositions and methods
AU2022325232A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified primary cells for allogeneic cell therapy
WO2025117331A1 (en) 2023-12-01 2025-06-05 Eli Lilly And Company Methods of making stem cell-derived islet-like cells, as well as populations and compositions including the same
CN118291605A (zh) * 2024-04-10 2024-07-05 深圳市血液中心(深圳市输血医学研究所) 一种基于ngs的多系统红细胞血型基因分型方法和试剂盒
CN119913243A (zh) * 2025-04-01 2025-05-02 浙江省血液中心 Abo血型变异体的snp位点、b亚型变异型血型鉴定试剂盒和方法

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB8724885D0 (en) 1987-10-23 1987-11-25 Binns M M Fowlpox virus promotors
DE19539493A1 (de) 1995-10-24 1997-04-30 Thomae Gmbh Dr K Starker homologer Promotor aus Hamster
CA3111953C (en) 2011-04-05 2023-10-24 Cellectis Method for the generation of compact tale-nucleases and uses thereof
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
IL239344B2 (en) 2012-12-12 2024-06-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
CN113355357B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CN107921148A (zh) 2015-05-08 2018-04-17 哈佛学院校长同事会 通用供体干细胞和相关方法
WO2017194699A1 (en) * 2016-05-13 2017-11-16 University Of Copenhagen A cell-based array platform
JP2020505025A (ja) * 2017-01-13 2020-02-20 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 免疫工学的な改変をした多能性細胞
IL270835B2 (en) 2017-06-12 2025-01-01 Sinai Health Sys Allograft tolerance without the need for systemic immunosuppression
WO2019084531A1 (en) * 2017-10-27 2019-05-02 The Children's Hospital Of Philadelphia MODIFIED RED GLOBES HAVING PHENOTYPES OF RARE ANTIGENS
WO2020018620A1 (en) * 2018-07-17 2020-01-23 The Regents Of The University Of California Chimeric antigen receptor t cells derived from immunoengineered pluripotent stem cells
CA3109078A1 (en) * 2018-07-17 2020-01-23 The Regents Of The University Of California Cells differentiated from immunoengineered pluripotent cells
US11162079B2 (en) * 2019-05-10 2021-11-02 The Regents Of The University Of California Blood type O Rh-hypo-immunogenic pluripotent cells
US12221622B2 (en) * 2019-05-10 2025-02-11 The Regents Of The University Of California Modified pluripotent cells

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118638907A (zh) * 2024-06-26 2024-09-13 中国人民解放军联勤保障部队第九六〇医院 一种用于检测易漏检类孟买表型的snp标记

Also Published As

Publication number Publication date
US20230272429A1 (en) 2023-08-31
JP2023510872A (ja) 2023-03-15
JP7853210B2 (ja) 2026-04-28
WO2021146222A1 (en) 2021-07-22
EP4090743A1 (en) 2022-11-23
CA3164591A1 (en) 2021-07-22
AU2021207810A1 (en) 2022-08-04

Similar Documents

Publication Publication Date Title
US20230272429A1 (en) Modification of blood type antigens
US11977073B2 (en) HLA G-modified cells and methods
JP2025069169A (ja) 免疫工学的な改変をした多能性細胞
JP6933898B2 (ja) 養子細胞治療製品を製造するための誘導多能性幹細胞の適用
KR20220007093A (ko) 변형된 만능 세포
JP2023510916A (ja) 遺伝子発現の制御のための安全スイッチ
JP2021530999A (ja) 免疫改変多能性幹細胞由来のキメラ抗原受容体ｔ細胞
KR20220082045A (ko) Fc 격리를 통한 이식된 세포 보호
JP2021515576A (ja) フォリスタチンを分泌するように遺伝子操作されたｂ細胞ならびにフォリスタチン関連疾患、状態、障害を処置するために、ならびに筋肉の成長および強度を増強するためにこれを使用する方法
IL323652A (en) Stem cells that are used for memorialization and their uses
EP4585223A2 (en) Transplanted cell protection via inhibition of polymorphonuclear cells
CN118076362A (zh) 用于改变低免疫原性细胞中的基因表达的诱导型系统
HK40066059A (en) Hla-g transfected cells and their use in treating diabetes

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination