CA3067382A1 - Targeted non-viral dna insertions - Google Patents

Targeted non-viral dna insertions Download PDF

Info

Publication number
CA3067382A1
CA3067382A1 CA3067382A CA3067382A CA3067382A1 CA 3067382 A1 CA3067382 A1 CA 3067382A1 CA 3067382 A CA3067382 A CA 3067382A CA 3067382 A CA3067382 A CA 3067382A CA 3067382 A1 CA3067382 A1 CA 3067382A1
Authority
CA
Canada
Prior art keywords
cell
cells
rnp
dna template
gfp
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
CA3067382A
Other languages
English (en)
French (fr)
Inventor
Theodore Lee ROTH
Alexander Marson
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.)
University of California San Diego UCSD
Original Assignee
University of California San Diego UCSD
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 University of California San Diego UCSD filed Critical University of California San Diego UCSD
Publication of CA3067382A1 publication Critical patent/CA3067382A1/en
Pending legal-status Critical Current

Links

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
    • 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
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0636—T lymphocytes
    • 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]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0016—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing 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
    • 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
    • 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
    • C12N15/1138—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
    • 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
    • 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
    • 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]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • C12N9/226—Class 2 CAS enzyme complex, e.g. single CAS protein
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hematology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3067382A 2017-06-15 2018-06-15 Targeted non-viral dna insertions Pending CA3067382A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762520117P 2017-06-15 2017-06-15
US62/520,117 2017-06-15
US201762552180P 2017-08-30 2017-08-30
US62/552,180 2017-08-30
PCT/US2018/037919 WO2018232356A1 (en) 2017-06-15 2018-06-15 Targeted non-viral dna insertions

Publications (1)

Publication Number Publication Date
CA3067382A1 true CA3067382A1 (en) 2018-12-20

Family

ID=64659467

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3067382A Pending CA3067382A1 (en) 2017-06-15 2018-06-15 Targeted non-viral dna insertions

Country Status (13)

Country Link
US (5) US20200362355A1 (https=)
EP (1) EP3638317A4 (https=)
JP (3) JP7275054B2 (https=)
KR (3) KR20260023091A (https=)
CN (2) CN118599915A (https=)
AU (2) AU2018283405B2 (https=)
BR (1) BR112019026625A2 (https=)
CA (1) CA3067382A1 (https=)
IL (1) IL271389B2 (https=)
MX (3) MX2019015188A (https=)
MY (1) MY200418A (https=)
SG (1) SG11201912179SA (https=)
WO (1) WO2018232356A1 (https=)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018232356A1 (en) 2017-06-15 2018-12-20 The Regents Of The University Of California Targeted non-viral dna insertions
JP7101419B2 (ja) 2017-10-27 2022-07-15 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 内因性ｔ細胞受容体の標的置換
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
US20230158110A1 (en) * 2018-05-30 2023-05-25 The Regents Of The University Of California Gene editing of monogenic disorders in human hematopoietic stem cells -- correction of x-linked hyper-igm syndrome (xhim)
CN113825834A (zh) * 2019-03-11 2021-12-21 索伦托药业有限公司 使用rna引导的核酸内切酶进行dna构建体整合的改进工艺
CN114107292B (zh) * 2020-08-27 2024-03-12 阿思科力(苏州)生物科技有限公司 用于外源基因定点插入的基因编辑系统和方法
CN116529385A (zh) * 2020-08-27 2023-08-01 天美治疗公司 用于制造工程化免疫细胞的无载体方法
CN113046381A (zh) * 2021-04-12 2021-06-29 南华大学 用于分离生物体内特异性蛋白-dna复合体的方法、融合蛋白及其制备方法
JP2024537991A (ja) 2021-10-14 2024-10-18 アーセナル バイオサイエンシズ インコーポレイテッド 共発現されるｓｈＲＮＡと論理ゲートシステムとを有する免疫細胞
US20250305003A1 (en) * 2022-05-10 2025-10-02 Editas Medicine, Inc. Genome editing of cells
PE20251319A1 (es) 2022-09-13 2025-05-16 Arsenal Biosciences Inc Celulas inmunitarias que coexpresan arnhc de tgfbr
EP4587570A2 (en) 2022-09-16 2025-07-23 Arsenal Biosciences, Inc. Immune cells with combination gene perturbations
AU2024228388A1 (en) 2023-02-27 2025-10-02 Adaptive Biotechnologies Corp. Therapeutic t cell receptors targeting kras g12d
IL322949A (en) 2023-03-03 2025-10-01 Arsenal Biosciences Inc Systems targeting PSMA and CA9
IL322838A (en) 2023-03-13 2025-10-01 Arsenal Biosciences Inc Synthetic pathway operators
AU2024303521A1 (en) * 2023-06-14 2026-01-15 Arsenal Biosciences, Inc. Non-viral cell engineering
WO2025199346A1 (en) 2024-03-20 2025-09-25 Arsenal Biosciences, Inc. Antigen binding proteins that bind tmprss4 and methods of use thereof
US20250297255A1 (en) 2024-03-20 2025-09-25 Arsenal Biosciences, Inc. Systems targeting tmprss4 and slc34a2
WO2026055342A1 (en) 2024-09-04 2026-03-12 Arsenal Biosciences, Inc. Synthetic pathway activators

Family Cites Families (111)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6773669B1 (en) 1995-03-10 2004-08-10 Maxcyte, Inc. Flow electroporation chamber and method
US6090617A (en) 1996-12-05 2000-07-18 Entremed, Inc. Flow electroporation chamber with electrodes having a crystalline metal nitride coating
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
CA2407695C (en) 2000-04-28 2015-03-31 Sangamo Biosciences, Inc. Methods for binding an exogenous molecule to cellular chromatin
US7029916B2 (en) 2001-02-21 2006-04-18 Maxcyte, Inc. Apparatus and method for flow electroporation of biological samples
CN1302115C (zh) 2001-04-23 2007-02-28 埃麦克萨有限公司 电穿孔用缓冲溶液及上述溶液的使用方法
AU2002326717A1 (en) 2001-08-22 2003-03-10 Maxcyte, Inc. Apparatus and method for electroporation of biological samples
GB0124391D0 (en) 2001-10-11 2001-11-28 Gene Expression Technologies L Control of gene expression
US20050136040A1 (en) 2001-10-11 2005-06-23 Imperial College Innovations Limited Control of gene expression using a complex of an oligonucleotide and a regulatory peptide
WO2003080809A2 (en) 2002-03-21 2003-10-02 Sangamo Biosciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
ITMI20030821A1 (it) 2003-04-18 2004-10-19 Internat Ct For Genetic En Gineering And Polipeptidi chimerici e loro uso.
US20060182736A1 (en) 2003-06-10 2006-08-17 Kim Jin-Soo Transducible dna-binding proteins
US8133733B2 (en) 2003-10-24 2012-03-13 Gencia Corporation Nonviral vectors for delivering polynucleotides to target tissues
DE502004009820D1 (de) 2004-03-15 2009-09-10 Lonza Cologne Ag Behältnis und Vorrichtung zur Erzeugung von elektrischen Feldern in einzelnen Reaktionsräumen
US7771984B2 (en) 2004-05-12 2010-08-10 Maxcyte, Inc. Methods and devices related to a regulated flow electroporation chamber
JP4519915B2 (ja) 2004-06-12 2010-08-04 インビトロゲン シンガポール ピーティーイー．リミテッド． 中空構造を有する電気穿孔装置
ES2465467T3 (es) 2004-06-14 2014-06-05 Lonza Cologne Ag Procedimiento y disposición de circuito para el tratamiento de material biológico
US20070254291A1 (en) 2004-06-14 2007-11-01 Xiaoxia Cui Gene Targeting in Eukaryotic Cells by Group II Intron Ribonucleoprotein Particles
US7991559B2 (en) 2004-11-30 2011-08-02 Maxcyte Inc. Computerized electroporation
US10022457B2 (en) 2005-08-05 2018-07-17 Gholam A. Peyman Methods to regulate polarization and enhance function of cells
DK2341149T3 (en) 2005-08-26 2017-02-27 Dupont Nutrition Biosci Aps Use of CRISPR-associated genes (Cas)
BRPI0716427A2 (pt) 2006-08-11 2014-03-11 Dow Agrosciences Llc Recombinação homóloga mediada por nuclease de apêndice de zinco
US8450112B2 (en) 2008-04-09 2013-05-28 Maxcyte, Inc. Engineering and delivery of therapeutic compositions of freshly isolated cells
US20120088842A1 (en) 2008-07-18 2012-04-12 Maxcyte, Inc. Methods for optimizing electroporation
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
EP2424877A4 (en) 2009-04-28 2013-01-02 Harvard College SUPRAGELADENE PROTEINS FOR CELL PENETRATION
WO2011059836A2 (en) 2009-10-29 2011-05-19 Trustees Of Dartmouth College T cell receptor-deficient t cell compositions
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
WO2011072246A2 (en) 2009-12-10 2011-06-16 Regents Of The University Of Minnesota Tal effector-mediated dna modification
US9567573B2 (en) 2010-04-26 2017-02-14 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using nucleases
US8876458B2 (en) 2011-01-25 2014-11-04 United Technologies Corporation Blade outer air seal assembly and support
US10391126B2 (en) 2011-11-18 2019-08-27 Board Of Regents, The University Of Texas System CAR+ T cells genetically modified to eliminate expression of T-cell receptor and/or HLA
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
US20130236504A1 (en) 2012-03-06 2013-09-12 Medical University Of South Carolina Delivery System for Enhancing Drug Efficacy
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
AU2013335451C1 (en) 2012-10-23 2024-07-04 Toolgen Incorporated Composition for cleaving a target DNA comprising a guide RNA specific for the target DNA and Cas protein-encoding nucleic acid or Cas protein, and use thereof
PL3360964T3 (pl) 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
DK2931891T3 (da) 2012-12-17 2019-08-19 Harvard College Rna-styret modificering af menneskelige genomer
KR20140101203A (ko) 2013-02-08 2014-08-19 이정민 자동 버핑장치
CN112301024A (zh) 2013-03-15 2021-02-02 通用医疗公司 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性
US9937207B2 (en) 2013-03-21 2018-04-10 Sangamo Therapeutics, Inc. Targeted disruption of T cell receptor genes using talens
CA2908403A1 (en) 2013-04-02 2014-10-09 Bayer Cropscience Nv Targeted genome engineering in eukaryotes
WO2014191128A1 (en) 2013-05-29 2014-12-04 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
ES2883131T3 (es) 2013-05-29 2021-12-07 Cellectis Métodos para la modificación de células T para inmunoterapia utilizando el sistema de nucleasa CAS guiado por ARN
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
WO2015048690A1 (en) 2013-09-27 2015-04-02 The Regents Of The University Of California Optimized small guide rnas and methods of use
EP3441468B1 (en) 2013-10-17 2021-05-19 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
CA2930590C (en) 2013-11-15 2021-02-16 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Engineering neural stem cells using homologous recombination
US10787684B2 (en) * 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
EP3470089A1 (en) 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
JP6712948B2 (ja) * 2013-12-12 2020-06-24 ザ・ブロード・インスティテュート・インコーポレイテッド 組成物、及びヌクレオチドリピート障害におけるｃｒｉｓｐｒ−ｃａｓ系の使用方法
JP7103750B2 (ja) 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用
US20160304893A1 (en) 2013-12-13 2016-10-20 Cellectis Cas9 nuclease platform for microalgae genome engineering
EP3099801B1 (en) 2014-01-31 2020-03-18 Factor Bioscience Inc. Synthetic rna for use in the treatment of dystrophic epidermolysis bullosa
WO2015115903A1 (en) 2014-02-03 2015-08-06 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Site-specific dna break-induced genome editing using engineered nucleases
WO2015161276A2 (en) 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
EP3155116A4 (en) 2014-06-10 2017-12-27 Massachusetts Institute Of Technology Method for gene editing
JP6598860B2 (ja) 2014-08-06 2019-10-30 カレッジ オブ メディシン ポチョン チャ ユニバーシティ インダストリー−アカデミック コーオペレイション ファウンデーション Ｈｌａをコードする遺伝子のヌクレアーゼ媒介編集により作製される免疫適合性細胞
EP3188763B1 (en) 2014-09-02 2020-05-13 The Regents of The University of California Methods and compositions for rna-directed target dna modification
WO2016049251A1 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes
US9879283B2 (en) 2014-10-09 2018-01-30 Life Technologies Corporation CRISPR oligonucleotides and gene editing
US20170335331A1 (en) 2014-10-31 2017-11-23 The Trustees Of The University Of Pennsylvania Altering Gene Expression in CART Cells and Uses Thereof
WO2016097751A1 (en) 2014-12-18 2016-06-23 The University Of Bath Method of cas9 mediated genome engineering
JP2018509387A (ja) 2015-01-21 2018-04-05 フェーズアールエックス インコーポレイテッド 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム
WO2016123578A1 (en) 2015-01-30 2016-08-04 The Regents Of The University Of California Protein delivery in primary hematopoietic cells
WO2016135559A2 (en) 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of human genetic diseases including hemoglobinopathies
CN107614012A (zh) 2015-04-24 2018-01-19 加利福尼亚大学董事会 使用工程化的细胞检测、监测或治疗疾病或病况的系统及制备和使用它们的方法
WO2016196388A1 (en) 2015-05-29 2016-12-08 Juno Therapeutics, Inc. Composition and methods for regulating inhibitory interactions in genetically engineered cells
JP2018518182A (ja) 2015-06-17 2018-07-12 ザ ユーエービー リサーチ ファンデーション ゲノム編集のためのｃｒｉｓｐｒ／ｃａｓ９複合体
JP2018518181A (ja) * 2015-06-17 2018-07-12 ザ ユーエービー リサーチ ファンデーション 血液細胞系列の細胞に機能的ポリペプチドを導入するためのＣＲＩＳＰＲ／Ｃａｓ９複合体
US20170000743A1 (en) 2015-07-02 2017-01-05 Vindico NanoBio Technology Inc. Compositions and Methods for Delivery of Gene Editing Tools Using Polymeric Vesicles
AU2016291778B2 (en) 2015-07-13 2021-05-06 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US20180243446A1 (en) 2015-09-01 2018-08-30 The Hospital For Sick Children Method and compositions for removing duplicated copy number variaions (cnvs) for genetic disorders and related uses
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
EP3356533A1 (en) 2015-09-28 2018-08-08 North Carolina State University Methods and compositions for sequence specific antimicrobials
JP6816133B2 (ja) 2015-10-05 2021-01-20 プレシジョン バイオサイエンシズ，インク． 改変ヒトｔ細胞受容体アルファ定常領域遺伝子を含む遺伝子改変細胞
US20180327706A1 (en) 2015-10-19 2018-11-15 The Methodist Hospital Crispr-cas9 delivery to hard-to-transfect cells via membrane deformation
EP3365454A1 (en) 2015-10-20 2018-08-29 10X Genomics, Inc. Methods and systems for high throughput single cell genetic manipulation
WO2017070429A1 (en) 2015-10-22 2017-04-27 Regents Of The University Of Minnesota Methods involving editing polynucleotides that encode t cell receptor
KR20250141836A (ko) 2015-11-04 2025-09-29 페이트 세러퓨틱스, 인코포레이티드 만능 세포의 유전자 조작
CA3005968A1 (en) 2015-11-23 2017-06-01 The Regents Of The University Of California Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9
WO2017093969A1 (en) * 2015-12-04 2017-06-08 Novartis Ag Compositions and methods for immunooncology
EP3389677B1 (en) 2015-12-18 2024-06-26 Sangamo Therapeutics, Inc. Targeted disruption of the t cell receptor
CA3009715A1 (en) 2015-12-30 2017-07-06 Avectas Limited Vector-free delivery of gene editing proteins and compositions to cells and tissues
JP2019509738A (ja) 2016-03-11 2019-04-11 ブルーバード バイオ， インコーポレイテッド ゲノム編集された免疫エフェクター細胞
US20190161530A1 (en) 2016-04-07 2019-05-30 Bluebird Bio, Inc. Chimeric antigen receptor t cell compositions
US10188749B2 (en) 2016-04-14 2019-01-29 Fred Hutchinson Cancer Research Center Compositions and methods to program therapeutic cells using targeted nucleic acid nanocarriers
EP4711465A2 (en) 2016-04-14 2026-03-18 Fred Hutchinson Cancer Center Compositions and methods to program therapeutic cells using targeted nucleic acid nanocarriers
EP4628587A3 (en) 2016-04-15 2026-02-25 Memorial Sloan Kettering Cancer Center Transgenic t cell and chimeric antigen receptor t cell compositions and related methods
US11499168B2 (en) * 2016-04-25 2022-11-15 Universitat Basel Allele editing and applications thereof
WO2017189336A1 (en) 2016-04-25 2017-11-02 The Regents Of The University Of California Methods and compositions for genomic editing
EP4166660A1 (en) 2016-04-29 2023-04-19 BASF Plant Science Company GmbH Improved methods for modification of target nucleic acids using fused guide rna - donor molecules
US20190136224A1 (en) 2016-05-31 2019-05-09 Massachusetts Institute Of Technology Hydrodynamically Controlled Electric Fields for High Throughput Transformation & High Throughput Parallel Transformation Platform
EP3472338B1 (en) 2016-06-20 2024-05-22 Octapharma AG Means and methods for modifying multiple alleles
WO2018035387A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
US11999931B2 (en) 2016-08-20 2024-06-04 The Regents Of The University Of California High-throughput system and method for the temporary permeabilization of cells
JP6837879B2 (ja) 2016-08-26 2021-03-03 日東電工株式会社 粘着テープ
CN110291100B (zh) 2016-10-12 2024-06-11 费尔丹生物公司 用于将多肽负荷从细胞外空间递送至靶真核细胞的胞质溶胶和/或细胞核的合理设计的合成肽穿梭剂，其用途、与其相关的方法和试剂盒
WO2018073391A1 (en) 2016-10-19 2018-04-26 Cellectis Targeted gene insertion for improved immune cells therapy
WO2018073393A2 (en) 2016-10-19 2018-04-26 Cellectis Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy
WO2018094291A1 (en) 2016-11-18 2018-05-24 Christopher Bradley Massively multiplexed homologous template repair for whole-genome replacement
AU2017378427A1 (en) 2016-12-14 2019-06-20 Ligandal, Inc. Methods and compositions for nucleic acid and protein payload delivery
CN110546495B (zh) 2016-12-30 2022-11-01 加利福尼亚州立大学董事会 用于基因组编辑t细胞的选择和传代的方法
WO2018232356A1 (en) 2017-06-15 2018-12-20 The Regents Of The University Of California Targeted non-viral dna insertions
JP7101419B2 (ja) 2017-10-27 2022-07-15 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 内因性ｔ細胞受容体の標的置換
SG11202004026QA (en) 2017-10-30 2020-05-28 Pact Pharma Inc Primary cell gene editing

Also Published As

Publication number Publication date
MY200418A (en) 2023-12-25
JP7275054B2 (ja) 2023-05-17
MX2024006956A (es) 2024-06-24
JP2023100828A (ja) 2023-07-19
AU2018283405A1 (en) 2020-01-16
BR112019026625A2 (pt) 2020-06-30
US20200362355A1 (en) 2020-11-19
JP2020524998A (ja) 2020-08-27
KR102720855B1 (ko) 2024-10-25
US20240117360A1 (en) 2024-04-11
KR20260023091A (ko) 2026-02-20
IL271389B2 (en) 2026-03-01
EP3638317A1 (en) 2020-04-22
US11814624B2 (en) 2023-11-14
US20210388362A1 (en) 2021-12-16
KR20200018645A (ko) 2020-02-19
US20220064653A1 (en) 2022-03-03
US12234461B2 (en) 2025-02-25
MX2025009289A (es) 2025-09-02
KR20240027153A (ko) 2024-02-29
SG11201912179SA (en) 2020-01-30
WO2018232356A1 (en) 2018-12-20
CN111344020A (zh) 2020-06-26
EP3638317A4 (en) 2021-03-17
IL271389A (en) 2020-01-30
AU2018283405B2 (en) 2025-06-26
AU2025238070A1 (en) 2025-10-23
JP2025078701A (ja) 2025-05-20
IL271389B1 (en) 2025-11-01
MX2019015188A (es) 2020-08-03
CN118599915A (zh) 2024-09-06
US20250320504A1 (en) 2025-10-16

Similar Documents

Publication Publication Date Title
US12234461B2 (en) Targeted non-viral DNA insertions
US12398193B2 (en) Targeted replacement of endogenous T cell receptors
HK40115875A (zh) 靶向非病毒dna插入
EA049987B1 (ru) Прицельная безвирусная вставка днк
TW202430630A (zh) 修飾的免疫細胞及其用途
EA050071B1 (ru) Направленная замена эндогенных т-клеточных рецепторов
HK40037128A (en) Targeted replacement of endogenous t cell receptors

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20230615

D15 Examination report completed

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT

Effective date: 20240702

D00 Search and/or examination requested or commenced

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D00-D120 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: VOLUNTARY SUBMISSION OF PRIOR ART RECEIVED

Effective date: 20241101

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION

Effective date: 20241101

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250410

D00 Search and/or examination requested or commenced

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D00-D123 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: PRIOR ART DISCLOSURE DETERMINED COMPLIANT

Effective date: 20250425

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20250425

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20250527

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20250527

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 7TH ANNIV.) - STANDARD

Year of fee payment: 7

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250606

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250606

D15 Examination report completed

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT

Effective date: 20250818

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION

Effective date: 20251218

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20260121

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20260121

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20260121