JP7275054B2 - 標的化された非ウイルスｄｎａ挿入 - Google Patents

標的化された非ウイルスｄｎａ挿入 Download PDF

Info

Publication number
JP7275054B2
JP7275054B2 JP2019569305A JP2019569305A JP7275054B2 JP 7275054 B2 JP7275054 B2 JP 7275054B2 JP 2019569305 A JP2019569305 A JP 2019569305A JP 2019569305 A JP2019569305 A JP 2019569305A JP 7275054 B2 JP7275054 B2 JP 7275054B2
Authority
JP
Japan
Prior art keywords
cells
rnp
dna template
cell
template
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.)
Active
Application number
JP2019569305A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2018232356A5 (https=
JP2020524998A (ja
JP2020524998A5 (https=
Inventor
セオドア リー ロス
アレクサンダー マーソン
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 JP2020524998A publication Critical patent/JP2020524998A/ja
Publication of JPWO2018232356A5 publication Critical patent/JPWO2018232356A5/ja
Publication of JP2020524998A5 publication Critical patent/JP2020524998A5/ja
Priority to JP2023076182A priority Critical patent/JP2023100828A/ja
Application granted granted Critical
Publication of JP7275054B2 publication Critical patent/JP7275054B2/ja
Priority to JP2025031513A priority patent/JP2025078701A/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • 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)
JP2019569305A 2017-06-15 2018-06-15 標的化された非ウイルスｄｎａ挿入 Active JP7275054B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023076182A JP2023100828A (ja) 2017-06-15 2023-05-02 標的化された非ウイルスｄｎａ挿入
JP2025031513A JP2025078701A (ja) 2017-06-15 2025-02-28 標的化された非ウイルスｄｎａ挿入

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023076182A Division JP2023100828A (ja) 2017-06-15 2023-05-02 標的化された非ウイルスｄｎａ挿入

Publications (4)

Publication Number Publication Date
JP2020524998A JP2020524998A (ja) 2020-08-27
JPWO2018232356A5 JPWO2018232356A5 (https=) 2022-02-07
JP2020524998A5 JP2020524998A5 (https=) 2022-02-07
JP7275054B2 true JP7275054B2 (ja) 2023-05-17

Family

ID=64659467

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2019569305A Active JP7275054B2 (ja) 2017-06-15 2018-06-15 標的化された非ウイルスｄｎａ挿入
JP2023076182A Pending JP2023100828A (ja) 2017-06-15 2023-05-02 標的化された非ウイルスｄｎａ挿入
JP2025031513A Pending JP2025078701A (ja) 2017-06-15 2025-02-28 標的化された非ウイルスｄｎａ挿入

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2023076182A Pending JP2023100828A (ja) 2017-06-15 2023-05-02 標的化された非ウイルスｄｎａ挿入
JP2025031513A Pending JP2025078701A (ja) 2017-06-15 2025-02-28 標的化された非ウイルスｄｎａ挿入

Country Status (13)

Country Link
US (5) US20200362355A1 (https=)
EP (1) EP3638317A4 (https=)
JP (3) JP7275054B2 (https=)
KR (3) KR20260023091A (https=)
CN (2) CN111344020A (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
CA3067382A1 (en) 2017-06-15 2018-12-20 The Regents Of The University Of California Targeted non-viral dna insertions
KR102503130B1 (ko) 2017-10-27 2023-02-24 더 리전트 오브 더 유니버시티 오브 캘리포니아 내인성 t 세포 수용체의 표적화된 대체
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
EP3833680A4 (en) * 2018-05-30 2022-05-25 The Regents of the University of California GENE EDIT OF MONOGENIC DISEASES IN HUMAN HEMATOPOIETIC STEM CELLS - CORRECTION OF X-LINKED HYPER-IGM SYNDROME (XHIM)
US20220145333A1 (en) * 2019-03-11 2022-05-12 Sorrento Therapeutics, Inc. Improved process for integration of dna constructs using rna-guided endonucleases
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
AU2022366987A1 (en) 2021-10-14 2024-05-16 Arsenal Biosciences, Inc. Immune cells having co-expressed shrnas and logic gate systems
EP4522182A2 (en) * 2022-05-10 2025-03-19 Editas Medicine, Inc. Genome editing of cells
KR20250068649A (ko) 2022-09-13 2025-05-16 아스널 바이오사이언시스, 인크. 공동 발현된 tgfbr shrna를 갖는 면역 세포
EP4587570A2 (en) 2022-09-16 2025-07-23 Arsenal Biosciences, Inc. Immune cells with combination gene perturbations
EP4673167A1 (en) 2023-02-27 2026-01-07 Adaptive Biotechnologies Corp. Therapeutic t cell receptors targeting kras g12d
KR20250158048A (ko) 2023-03-03 2025-11-05 아스널 바이오사이언시스, 인크. Psma 및 ca9을 표적으로 하는 시스템
PE20252678A1 (es) 2023-03-13 2025-11-24 Arsenal Biosciences Inc Activadores de vias sinteticas
EP4720316A2 (en) * 2023-06-14 2026-04-08 Arsenal Biosciences, Inc. Non-viral cell engineering
WO2025199338A1 (en) 2024-03-20 2025-09-25 Arsenal Biosciences, Inc. Systems targeting slc34a2 and tmprss4 and methods of use thereof
WO2025199346A1 (en) 2024-03-20 2025-09-25 Arsenal Biosciences, Inc. Antigen binding proteins that bind tmprss4 and methods of use thereof
WO2026055342A1 (en) 2024-09-04 2026-03-12 Arsenal Biosciences, Inc. Synthetic pathway activators

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016123578A1 (en) 2015-01-30 2016-08-04 The Regents Of The University Of California Protein delivery in primary hematopoietic cells
WO2016205680A1 (en) 2015-06-17 2016-12-22 The Uab Research Foundation Crispr/cas9 complex for introducing a functional polypeptide into cells of blood cell lineage

Family Cites Families (109)

* 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
WO2001083751A2 (en) 2000-04-28 2001-11-08 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
IL158514A0 (en) 2001-04-23 2004-05-12 Amaxa Gmbh Buffer solution for electroporation and a method comprising the use of the same
EP2574662B1 (en) 2001-08-22 2021-08-04 Maxcyte, Inc. 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
US20030232410A1 (en) 2002-03-21 2003-12-18 Monika Liljedahl 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.
WO2004108883A2 (en) 2003-06-10 2004-12-16 Toolgen, Inc. 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
DK1766057T3 (en) 2004-05-12 2015-03-02 Maxcyte Inc Methods and devices related to a regulated flowelektroporationskammer
KR100853102B1 (ko) 2004-06-12 2008-08-21 주식회사 디지탈바이오테크놀러지 중공구조를 갖는 전기천공 장치
EP1774036A4 (en) 2004-06-14 2008-10-15 Univ Texas At Austin GEN-TARGETING IN EUKARYOTIC CELLS BY GROUP II INTRON-RIBONUCLEOPROTEIN PARTICLES
ES2465467T3 (es) 2004-06-14 2014-06-05 Lonza Cologne Ag Procedimiento y disposición de circuito para el tratamiento de material biológico
KR20070091163A (ko) 2004-11-30 2007-09-07 맥스시티 인코포레이티드 컴퓨터화된 일렉트로포레이션
US10022457B2 (en) 2005-08-05 2018-07-17 Gholam A. Peyman Methods to regulate polarization and enhance function of cells
ES2398918T3 (es) 2005-08-26 2013-03-22 Dupont Nutrition Biosciences Aps Un método y una ordenación para soportar verticalmente elementos de resistencia eléctrica pendientes
SI2049663T1 (sl) 2006-08-11 2015-12-31 Dow Agrosciences Llc Homologna rekombinacija, posredovana z nukleazo s cinkovim prstom
US8450112B2 (en) 2008-04-09 2013-05-28 Maxcyte, Inc. Engineering and delivery of therapeutic compositions of freshly isolated cells
DK2310500T3 (en) 2008-07-18 2015-05-11 Maxcyte Inc Process for optimizing electroporation
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
JP2012525146A (ja) 2009-04-28 2012-10-22 プレジデント アンド フェロウズ オブ ハーバード カレッジ 細胞透過のための過剰に荷電されたタンパク質
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
HUE041436T2 (hu) 2009-12-10 2019-05-28 Univ Minnesota Tal-effektor-közvetített DNS-módosítás
US8771985B2 (en) 2010-04-26 2014-07-08 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using zinc-finger nucleases
US8876458B2 (en) 2011-01-25 2014-11-04 United Technologies Corporation Blade outer air seal assembly and support
WO2013074916A1 (en) 2011-11-18 2013-05-23 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
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
EP4357457B1 (en) 2012-10-23 2024-10-16 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
EP3363902B1 (en) 2012-12-06 2019-11-27 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
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
AU2013359262C1 (en) 2012-12-12 2021-05-13 Massachusetts Institute Of Technology CRISPR-Cas component systems, methods and compositions for sequence manipulation
CA2895155C (en) 2012-12-17 2021-07-06 President And Fellows Of Harvard College Rna-guided human genome engineering
KR20140101203A (ko) 2013-02-08 2014-08-19 이정민 자동 버핑장치
AU2014227653B2 (en) 2013-03-15 2017-04-20 The General Hospital Corporation Using RNA-guided foki nucleases (RFNs) to increase specificity for RNA-guided genome editing
JP6346266B2 (ja) 2013-03-21 2018-06-20 サンガモ セラピューティクス， インコーポレイテッド 操作されたジンクフィンガータンパク質ヌクレアーゼを使用するｔ細胞受容体遺伝子の標的化された破壊
EP2981614A1 (en) 2013-04-02 2016-02-10 Bayer CropScience NV Targeted genome engineering in eukaryotes
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
WO2014191128A1 (en) 2013-05-29 2014-12-04 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US10822606B2 (en) 2013-09-27 2020-11-03 The Regents Of The University Of California Optimized small guide RNAs and methods of use
CN105899665B (zh) 2013-10-17 2019-10-22 桑格摩生物科学股份有限公司 用于核酸酶介导的基因组工程改造的递送方法和组合物
WO2015073867A1 (en) 2013-11-15 2015-05-21 The United States Of America, As Represented By The Secretary, Department Of Health & 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
MX2016007325A (es) * 2013-12-12 2017-07-19 Broad Inst Inc Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.
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
AU2014361781B2 (en) 2013-12-12 2021-04-01 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing
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
CA2933134A1 (en) 2013-12-13 2015-06-18 Cellectis Cas9 nuclease platform for microalgae genome engineering
ES2939542T3 (es) 2014-01-31 2023-04-24 Factor Bioscience Inc Métodos y productos para la producción y la administración de ácido nucleico
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
CA2945335A1 (en) 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
WO2015191693A2 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
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
WO2016036754A1 (en) 2014-09-02 2016-03-10 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
WO2016057951A2 (en) 2014-10-09 2016-04-14 Life Technologies Corporation Crispr oligonucleotides and gene editing
WO2016069282A1 (en) 2014-10-31 2016-05-06 The Trustees Of The University Of Pennsylvania Altering gene expression in modified t cells and uses thereof
WO2016097751A1 (en) 2014-12-18 2016-06-23 The University Of Bath Method of cas9 mediated genome engineering
US11219634B2 (en) 2015-01-21 2022-01-11 Genevant Sciences Gmbh Methods, compositions, and systems for delivering therapeutic and diagnostic agents into 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
JP6949728B2 (ja) 2015-05-29 2021-10-13 ジュノー セラピューティクス インコーポレイテッド 遺伝子操作された細胞における阻害相互作用を調節するための組成物および方法
US11643668B2 (en) 2015-06-17 2023-05-09 The Uab Research Foundation CRISPR/Cas9 complex for genomic editing
US20170000743A1 (en) 2015-07-02 2017-01-05 Vindico NanoBio Technology Inc. Compositions and Methods for Delivery of Gene Editing Tools Using Polymeric Vesicles
EP3322297B1 (en) 2015-07-13 2024-12-04 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
WO2017035659A1 (en) 2015-09-01 2017-03-09 The Hospital For Sick Children Method and compositions for removing duplicated copy number variations (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
WO2017058751A1 (en) 2015-09-28 2017-04-06 North Carolina State University Methods and compositions for sequence specific antimicrobials
CN117070468A (zh) 2015-10-05 2023-11-17 精密生物科学公司 包含经修饰的人T细胞受体α恒定区基因的经遗传修饰的细胞
US20180327706A1 (en) 2015-10-19 2018-11-15 The Methodist Hospital Crispr-cas9 delivery to hard-to-transfect cells via membrane deformation
US20180312873A1 (en) 2015-10-20 2018-11-01 10X Genomics, Inc. Method 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
JP6928604B2 (ja) 2015-11-04 2021-09-01 フェイト セラピューティクス，インコーポレイテッド 万能性細胞のゲノム改変
JP2019500899A (ja) 2015-11-23 2019-01-17 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 ＣＲＩＳＰＲ／Ｃａｓ９の核送達を通じた細胞ＲＮＡの追跡と操作
EA201891338A1 (ru) * 2015-12-04 2018-12-28 Новартис Аг Композиции и способы для иммуноонкологии
AU2016369490C1 (en) 2015-12-18 2021-12-23 Sangamo Therapeutics, Inc. Targeted disruption of the T cell receptor
WO2017115128A2 (en) 2015-12-30 2017-07-06 Avectas Limited Vector-free delivery of gene editing proteins and compositions to cells and tissues
MX2018010924A (es) 2016-03-11 2019-02-13 Bluebird Bio Inc Células efectoras inmunitarias con edición genómica.
CN109415687A (zh) 2016-04-07 2019-03-01 蓝鸟生物公司 嵌合抗原受体t细胞组合物
AU2017250295B2 (en) 2016-04-14 2022-08-25 Fred Hutchinson Cancer Center Compositions and methods to program therapeutic cells using targeted nucleic acid nanocarriers
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
US11377637B2 (en) 2016-04-15 2022-07-05 Memorial Sloan Kettering Cancer Center Transgenic T cell and chimeric antigen receptor T cell compositions and related methods
WO2017189336A1 (en) 2016-04-25 2017-11-02 The Regents Of The University Of California Methods and compositions for genomic editing
US11499168B2 (en) * 2016-04-25 2022-11-15 Universitat Basel Allele editing and applications thereof
JP7184648B2 (ja) 2016-04-29 2022-12-06 ビーエーエスエフ プラント サイエンス カンパニー ゲーエムベーハー 標的核酸の改変のための改善された方法
WO2017210334A1 (en) 2016-05-31 2017-12-07 Massachusetts Institute Of Technology Hydrodynamically controlled electric fields for high throughput transformation & high throughput parallel transformation platform
ES2982085T3 (es) 2016-06-20 2024-10-14 Octapharma Ag Medios y métodos para modificar alelos múltiples
CN110312799A (zh) 2016-08-17 2019-10-08 博德研究所 新型crispr酶和系统
WO2018039084A1 (en) 2016-08-20 2018-03-01 The Regents Of The University Of California High-throughput system and method for the temporary permeablization of cells
JP6837879B2 (ja) 2016-08-26 2021-03-03 日東電工株式会社 粘着テープ
JP7177047B2 (ja) 2016-10-12 2022-11-22 フェルダン・バイオ・インコーポレーテッド ポリペプチドカーゴを標的真核細胞の細胞外間隙からサイトゾルおよび／または核に送達するための合理的に設計された合成ペプチドシャトル剤、その使用、それに関連する方法およびキット
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
EP3541937A4 (en) 2016-11-18 2020-06-24 Christopher Bradley REPAIR OF MASSIVELY MULTIPLEXED HOMOLOGICAL TEMPLATE FOR FULL GENERATION
CN110582301A (zh) 2016-12-14 2019-12-17 利甘达尔股份有限公司 用于核酸和蛋白质有效负载递送的方法和组合物
CA3048645A1 (en) 2016-12-30 2018-07-05 The Regents Of The University Of California Methods for selection and generation of genome edited t cells
CA3067382A1 (en) 2017-06-15 2018-12-20 The Regents Of The University Of California Targeted non-viral dna insertions
KR102503130B1 (ko) 2017-10-27 2023-02-24 더 리전트 오브 더 유니버시티 오브 캘리포니아 내인성 t 세포 수용체의 표적화된 대체
MX2020004541A (es) 2017-10-30 2021-04-12 Pact Pharma Inc Edicion de genes de celulas primarias.

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016123578A1 (en) 2015-01-30 2016-08-04 The Regents Of The University Of California Protein delivery in primary hematopoietic cells
WO2016205680A1 (en) 2015-06-17 2016-12-22 The Uab Research Foundation Crispr/cas9 complex for introducing a functional polypeptide into cells of blood cell lineage

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
J.Biotechnol.(2016.11)Vol.241, p.136-146
Nature，2018年07月11日，Vol.559，p.405-409

Also Published As

Publication number Publication date
BR112019026625A2 (pt) 2020-06-30
MX2019015188A (es) 2020-08-03
AU2018283405A1 (en) 2020-01-16
US20240117360A1 (en) 2024-04-11
CN111344020A (zh) 2020-06-26
AU2018283405B2 (en) 2025-06-26
US20220064653A1 (en) 2022-03-03
US20200362355A1 (en) 2020-11-19
AU2025238070A1 (en) 2025-10-23
MX2025009289A (es) 2025-09-02
CN118599915A (zh) 2024-09-06
US12234461B2 (en) 2025-02-25
KR102720855B1 (ko) 2024-10-25
KR20240027153A (ko) 2024-02-29
KR20200018645A (ko) 2020-02-19
JP2025078701A (ja) 2025-05-20
WO2018232356A1 (en) 2018-12-20
MX2024006956A (es) 2024-06-24
KR20260023091A (ko) 2026-02-20
JP2020524998A (ja) 2020-08-27
JP2023100828A (ja) 2023-07-19
US11814624B2 (en) 2023-11-14
SG11201912179SA (en) 2020-01-30
US20250320504A1 (en) 2025-10-16
IL271389B2 (en) 2026-03-01
US20210388362A1 (en) 2021-12-16
EP3638317A4 (en) 2021-03-17
MY200418A (en) 2023-12-25
CA3067382A1 (en) 2018-12-20
IL271389A (en) 2020-01-30
EP3638317A1 (en) 2020-04-22
IL271389B1 (en) 2025-11-01

Similar Documents

Publication Publication Date Title
JP7275054B2 (ja) 標的化された非ウイルスｄｎａ挿入
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
EA050071B1 (ru) Направленная замена эндогенных т-клеточных рецепторов
HK40037128A (en) Targeted replacement of endogenous t cell receptors

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200205

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20210125

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210615

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210615

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211227

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220705

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20221004

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221223

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20230404

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20230502

R150 Certificate of patent or registration of utility model

Ref document number: 7275054

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150