JP7575950B2 - 遺伝子編集されたcd34+細胞におけるfoxp3の発現 - Google Patents

遺伝子編集されたcd34+細胞におけるfoxp3の発現 Download PDF

Info

Publication number
JP7575950B2
JP7575950B2 JP2020560400A JP2020560400A JP7575950B2 JP 7575950 B2 JP7575950 B2 JP 7575950B2 JP 2020560400 A JP2020560400 A JP 2020560400A JP 2020560400 A JP2020560400 A JP 2020560400A JP 7575950 B2 JP7575950 B2 JP 7575950B2
Authority
JP
Japan
Prior art keywords
sequence
foxp3
cell
nucleic acid
nucleotides
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
JP2020560400A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021521856A (ja
JPWO2019210042A5 (enExample
Inventor
ジェイ. ローリングス,デイヴィッド
エフ. ハン,イラム
チアン ホナカー,ユーチ
シン,スワティ
ソマー,カレン
エム. シャーレンバーグ,アンドリュー
Original Assignee
シアトル チルドレンズ ホスピタル ディー/ビー/エイ シアトル チルドレンズ リサーチ インスティテュート
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 シアトル チルドレンズ ホスピタル ディー/ビー/エイ シアトル チルドレンズ リサーチ インスティテュート filed Critical シアトル チルドレンズ ホスピタル ディー/ビー/エイ シアトル チルドレンズ リサーチ インスティテュート
Publication of JP2021521856A publication Critical patent/JP2021521856A/ja
Publication of JPWO2019210042A5 publication Critical patent/JPWO2019210042A5/ja
Priority to JP2024182581A priority Critical patent/JP2025013884A/ja
Application granted granted Critical
Publication of JP7575950B2 publication Critical patent/JP7575950B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14Blood; Artificial blood
    • A61K35/15Cells of the myeloid line, e.g. granulocytes, basophils, eosinophils, neutrophils, leucocytes, monocytes, macrophages or mast cells; Myeloid precursor cells; Antigen-presenting cells, e.g. dendritic cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • C07K14/4703Inhibitors; Suppressors
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4713Autoimmune diseases, e.g. Insulin-dependent diabetes mellitus, multiple sclerosis, rheumathoid arthritis, systemic lupus erythematosus; Autoantigens
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0636T lymphocytes
    • C12N5/0637Immunosuppressive T lymphocytes, e.g. regulatory T cells or Treg
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0645Macrophages, e.g. Kuepfer cells in the liver; Monocytes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60Transcription factors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00Genetically modified cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors 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)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Diabetes (AREA)
  • Rehabilitation Therapy (AREA)
  • Rheumatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2020560400A 2018-04-27 2019-04-25 遺伝子編集されたcd34+細胞におけるfoxp3の発現 Active JP7575950B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024182581A JP2025013884A (ja) 2018-04-27 2024-10-18 遺伝子編集されたcd34+細胞におけるfoxp3の発現

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862663545P 2018-04-27 2018-04-27
US62/663,545 2018-04-27
PCT/US2019/029082 WO2019210042A1 (en) 2018-04-27 2019-04-25 Expression of foxp3 in edited cd34+ cells

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024182581A Division JP2025013884A (ja) 2018-04-27 2024-10-18 遺伝子編集されたcd34+細胞におけるfoxp3の発現

Publications (3)

Publication Number Publication Date
JP2021521856A JP2021521856A (ja) 2021-08-30
JPWO2019210042A5 JPWO2019210042A5 (enExample) 2022-11-25
JP7575950B2 true JP7575950B2 (ja) 2024-10-31

Family

ID=68294731

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020560400A Active JP7575950B2 (ja) 2018-04-27 2019-04-25 遺伝子編集されたcd34+細胞におけるfoxp3の発現
JP2024182581A Pending JP2025013884A (ja) 2018-04-27 2024-10-18 遺伝子編集されたcd34+細胞におけるfoxp3の発現

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024182581A Pending JP2025013884A (ja) 2018-04-27 2024-10-18 遺伝子編集されたcd34+細胞におけるfoxp3の発現

Country Status (9)

Country Link
US (2) US11713459B2 (enExample)
EP (1) EP3784690A4 (enExample)
JP (2) JP7575950B2 (enExample)
CN (1) CN112218882A (enExample)
AU (2) AU2019261438B2 (enExample)
CA (1) CA3091688A1 (enExample)
IL (1) IL277039A (enExample)
SG (1) SG11202007878UA (enExample)
WO (1) WO2019210042A1 (enExample)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2016427822B2 (en) 2016-10-31 2025-01-23 Seattle Children's Hospital (dba Seattle Children's Research Institute) Method for treating autoimmune disease using CD4 T-cells with engineered stabilization of expression of endogennous FOXP3 gene
CN112218882A (zh) * 2018-04-27 2021-01-12 西雅图儿童医院(Dba西雅图儿童研究所) Foxp3在经编辑的cd34+细胞中的表达
US20210253652A1 (en) * 2018-04-27 2021-08-19 Seattle Children's Hospital (dba Seattle Children's Research Institute) Expression of human foxp3 in gene edited t cells
EP3912644A4 (en) * 2019-01-18 2022-09-28 Osaka University THERAPEUTIC AGENT FOR DYSTROPHIC EPIDERMOLYSIS BULLOSA
CA3141159A1 (en) * 2019-05-21 2020-11-26 Sangamo Therapeutics, Inc. Controlled transgene expression in regulatory t cells
NZ783217A (en) * 2019-06-27 2025-08-29 Seattle Children’S Hospital Dba Seattle Children’S Res Institute Artificial antigen-specific immunoregulatory t (airt) cells
EP4055039A1 (en) 2019-11-08 2022-09-14 Sangamo Therapeutics, Inc. Generation of engineered regulatory t cells
WO2021144692A1 (en) * 2020-01-14 2021-07-22 Crispr Therapeutics Ag Methods for increased efficiency of homology-directed repair
WO2021163642A2 (en) * 2020-02-13 2021-08-19 The Board Of Trustees Of The Leland Stanford Junior University Crispr-based foxp3 gene engineered t cells and hematopoietic stem cell precursors to treat ipex syndrome patients
AU2021368557A1 (en) * 2020-10-27 2023-06-08 Adoc Ssf, Llc Compositions and methods for the treatment of cancer using next generation engineered t cell therapy
CN112851794B (zh) * 2021-02-04 2023-05-23 苏州铂维生物科技有限公司 一种基于cd271的抗原表位及其应用
GB202113674D0 (en) 2021-09-24 2021-11-10 Reflection Therapeutics Ltd Targeted cell therapies
GB202113673D0 (en) 2021-09-24 2021-11-10 Reflection Therapeutics Ltd Targeted cell therapies

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110123502A1 (en) 2007-02-21 2011-05-26 Barry Simon C Method for obtaining treg-cells
WO2016123578A1 (en) 2015-01-30 2016-08-04 The Regents Of The University Of California Protein delivery in primary hematopoietic cells
WO2018035141A1 (en) 2016-08-16 2018-02-22 Bluebird Bio, Inc. Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR100996016B1 (ko) 2000-09-20 2010-11-22 밀리포어 코포레이션 폴리뉴클레오티드
JP4353701B2 (ja) 2001-05-08 2009-10-28 ダーウィン モレキュラー コーポレイション Foxp3蛋白質を用いた霊長類における免疫機能の調節方法
GB0614536D0 (en) 2006-07-21 2006-08-30 Metcalfe Susan M Methods of controlling cellular response to LIF
EP2064350B1 (en) * 2006-11-27 2013-01-02 Ludwig Institute for Cancer Research Ltd. Expression of foxp3 by cancer cells
US20100135974A1 (en) 2007-01-31 2010-06-03 Yeda Research And Development Co. Ltd. Redirected, genetically-engineered t regulatory cells and their use in suppression of autoimmune and inflammatory disease
US9249423B2 (en) 2007-02-02 2016-02-02 Yale University Method of de-differentiating and re-differentiating somatic cells using RNA
WO2008141282A2 (en) 2007-05-11 2008-11-20 The Regents Of The University Of Michigan Materials and methods for foxp3 tumor suppression
WO2008154399A1 (en) 2007-06-08 2008-12-18 The Trustees Of The University Of Pennsylvania Foxp3 oligomerization and intermolecular interactions
JP2011503232A (ja) 2007-11-20 2011-01-27 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッド 免疫応答の調節
JP2011513403A (ja) 2008-03-03 2011-04-28 コンバージ バイオテック, インコーポレイテッド T細胞依存性免疫応答を調節する方法
WO2012018930A1 (en) 2010-08-03 2012-02-09 University Of Miami Methods of isolating and expanding human t regulatory cells and uses thereof for cellular therapy
WO2014180943A1 (en) 2013-05-08 2014-11-13 Vib Vzw Mcl-1 as critical regulator of foxp3+ regulatory t cell survival, and use thereof to treat severe immune disorders
AU2014266833B2 (en) 2013-05-13 2020-07-02 Cellectis Methods for engineering highly active T cell for immunotherapy
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
DK3004337T3 (da) 2013-05-29 2017-11-13 Cellectis Fremgangsmåde til konstruktion af T-celler til immunoterapi ved brug af RNA-guidet Cas nuklease-system
JP6673838B2 (ja) 2014-02-14 2020-04-01 セレクティスCellectis 免疫細胞と病的細胞の両方に存在する抗原を標的とするように操作された、免疫療法のための細胞
US20170198306A1 (en) 2014-03-21 2017-07-13 Cellectis Engineering mammalian genome using dna-guided argonaute interference systems (dais)
KR101835554B1 (ko) 2014-06-24 2018-04-19 서울대학교 산학협력단 C/ebp를 포함하는 유도성 t 조절세포 분화촉진 또는 안정화 조성물 및 방법
EP3245294A4 (en) 2015-01-12 2018-05-30 Massachusetts Institute of Technology Gene editing through microfluidic delivery
HK1254190A1 (zh) 2015-05-08 2019-07-12 President And Fellows Of Harvard College 通用供体干细胞和相关方法
US11499168B2 (en) 2016-04-25 2022-11-15 Universitat Basel Allele editing and applications thereof
WO2018031762A1 (en) 2016-08-10 2018-02-15 Duke University Compositions, systems and methods for programming immune cell function through targeted gene regulation
WO2018073391A1 (en) * 2016-10-19 2018-04-26 Cellectis Targeted gene insertion for improved immune cells therapy
WO2018081476A2 (en) 2016-10-27 2018-05-03 Intima Bioscience, Inc. Viral methods of t cell therapy
AU2016427822B2 (en) 2016-10-31 2025-01-23 Seattle Children's Hospital (dba Seattle Children's Research Institute) Method for treating autoimmune disease using CD4 T-cells with engineered stabilization of expression of endogennous FOXP3 gene
JP7206214B2 (ja) 2016-12-13 2023-01-17 シアトル チルドレンズ ホスピタル (ディービーエイ シアトル チルドレンズ リサーチ インスティテュート) インビトロ及びインビボで操作された細胞において発現された化学誘導シグナル伝達複合体の外因性薬物活性化の方法
CN109790518A (zh) 2017-05-08 2019-05-21 中国科学院动物研究所 经修饰的t细胞、其制备方法及用途
EP3672617A4 (en) * 2017-08-22 2021-06-23 The Regents of the University of California LENTIVIRAL VECTORS EXPRESSING FOXP3 IN HEMATOPOIETIC STEM CELLS TO TREAT IMMUNITY DEFICIENCIES AND AUTOIMMUNE DISEASES
CN112218882A (zh) * 2018-04-27 2021-01-12 西雅图儿童医院(Dba西雅图儿童研究所) Foxp3在经编辑的cd34+细胞中的表达
US20210253652A1 (en) 2018-04-27 2021-08-19 Seattle Children's Hospital (dba Seattle Children's Research Institute) Expression of human foxp3 in gene edited t cells
WO2019241549A1 (en) 2018-06-15 2019-12-19 A2 Biotherapeutics, Inc. Foxp3-expressing car-t regulatory cells

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110123502A1 (en) 2007-02-21 2011-05-26 Barry Simon C Method for obtaining treg-cells
WO2016123578A1 (en) 2015-01-30 2016-08-04 The Regents Of The University Of California Protein delivery in primary hematopoietic cells
WO2018035141A1 (en) 2016-08-16 2018-02-22 Bluebird Bio, Inc. Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
GOODWIN, Marianne et al.,123. Gene Editing as a Therapeutic Approach to Treat IPEX Stndrome,Molecular Therapy,2016年05月,Vol. 24, Supplement 1,p. S51,DOI:10.1016/S1525-0016(16)32932-X

Also Published As

Publication number Publication date
EP3784690A1 (en) 2021-03-03
EP3784690A4 (en) 2022-01-19
CA3091688A1 (en) 2019-10-31
SG11202007878UA (en) 2020-09-29
CN112218882A (zh) 2021-01-12
JP2025013884A (ja) 2025-01-28
AU2019261438A1 (en) 2020-09-10
AU2024266824A1 (en) 2024-12-12
JP2021521856A (ja) 2021-08-30
US20240117352A1 (en) 2024-04-11
IL277039A (en) 2020-10-29
US20210054376A1 (en) 2021-02-25
AU2019261438B2 (en) 2024-08-22
US11713459B2 (en) 2023-08-01
WO2019210042A1 (en) 2019-10-31

Similar Documents

Publication Publication Date Title
JP7575950B2 (ja) 遺伝子編集されたcd34+細胞におけるfoxp3の発現
JP7575949B2 (ja) 遺伝子編集t細胞におけるヒトfoxp3の発現
KR102532663B1 (ko) 베타 이상헤모글로빈증의 치료를 위한 crispr/cas-관련 방법 및 조성물
AU2016225179C1 (en) Materials and methods for treatment of hemoglobinopathies
KR102877920B1 (ko) 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법
JP7480062B2 (ja) ラパマイシン耐性細胞
KR102728416B1 (ko) A형 혈우병을 위한 유전자 편집용 조성물 및 방법
US20180119123A1 (en) Crispr/cas-related methods and compositions for treating hiv infection and aids
CN112020558A (zh) 治疗血红蛋白病的系统和方法
CA3085338A1 (en) Cpf1-related methods and compositions for gene editing
MD3445388T2 (ro) Materiale și metode pentru tratamentul hemoglobinopatiilor
EP3701028B1 (en) Systems and methods for treating hyper-igm syndrome
JP2024075603A (ja) Hla遺伝子のrnaガイドゲノム編集を使用して関節リウマチを処置する方法
US20200263206A1 (en) Targeted integration systems and methods for the treatment of hemoglobinopathies
CN114072518B (zh) 用于治疗地中海贫血或镰状细胞病的方法和组合物
US20240335561A1 (en) A method for in vivo gene therapy to cure scd without myeloablative toxicity
HK40069063B (zh) 用於治疗地中海贫血或镰状细胞病的方法和组合物

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210129

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220420

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220420

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221115

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230328

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230621

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20230822

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230927

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20231219

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240313

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240516

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: 20240820

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240913

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20241018

R150 Certificate of patent or registration of utility model

Ref document number: 7575950

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150