CN112218882A - Foxp3在经编辑的cd34+细胞中的表达 - Google Patents

Foxp3在经编辑的cd34+细胞中的表达 Download PDF

Info

Publication number
CN112218882A
CN112218882A CN201980028370.7A CN201980028370A CN112218882A CN 112218882 A CN112218882 A CN 112218882A CN 201980028370 A CN201980028370 A CN 201980028370A CN 112218882 A CN112218882 A CN 112218882A
Authority
CN
China
Prior art keywords
seq
cell
sequence
nucleic acid
foxp3
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
CN201980028370.7A
Other languages
English (en)
Chinese (zh)
Inventor
戴维德·J·罗林斯
伊拉姆·F·卡恩
玉池·蒋·霍纳克
斯瓦蒂·辛格
凯伦·索默
安德鲁·M·沙尔博格
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.)
Seattle Childrens Hospital
Original Assignee
Seattle Childrens Hospital
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 Seattle Childrens Hospital filed Critical Seattle Childrens Hospital
Publication of CN112218882A publication Critical patent/CN112218882A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—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
    • C12N15/87—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A61K35/15—Cells 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
    • 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
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4713—Autoimmune diseases, e.g. Insulin-dependent diabetes mellitus, multiple sclerosis, rheumathoid arthritis, systemic lupus erythematosus; Autoantigens
    • 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
    • C12N5/0637—Immunosuppressive T lymphocytes, e.g. regulatory T cells or Treg
    • 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
    • C12N5/0645—Macrophages, e.g. Kuepfer cells in the liver; Monocytes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • 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)
  • Diabetes (AREA)
  • Rehabilitation Therapy (AREA)
  • Rheumatology (AREA)
  • Mycology (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)
CN201980028370.7A 2018-04-27 2019-04-25 Foxp3在经编辑的cd34+细胞中的表达 Pending CN112218882A (zh)

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

Publications (1)

Publication Number Publication Date
CN112218882A true CN112218882A (zh) 2021-01-12

Family

ID=68294731

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201980028370.7A Pending CN112218882A (zh) 2018-04-27 2019-04-25 Foxp3在经编辑的cd34+细胞中的表达

Country Status (9)

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

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112851794A (zh) * 2021-02-04 2021-05-28 上海交通大学 一种基于cd271的新型抗原表位及其应用

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110139675B (zh) 2016-10-31 2023-09-29 西雅图儿童医院（Dba西雅图儿童研究所） 用具有工程化稳定的内源性foxp3基因表达的cd4t细胞治疗自身免疫疾病的方法
CA3091491A1 (en) 2018-04-27 2019-10-31 Seattle Children's Hospital (dba Seattle Children's Research Institute) Expression of human foxp3 in gene edited t cells
US11713459B2 (en) * 2018-04-27 2023-08-01 Seattle Children's Hospital Expression of FOXP3 in edited CD34+ cells
KR20210116531A (ko) * 2019-01-18 2021-09-27 고꾸리쯔 다이가꾸 호우징 오사까 다이가꾸 영양 장애형 표피 수포증 치료약
EP3973067A1 (en) * 2019-05-21 2022-03-30 Sangamo Therapeutics, Inc. Controlled transgene expression in regulatory t cells
AU2020304057A1 (en) * 2019-06-27 2022-01-27 Benaroya Research Institute At Virginia Mason Artificial antigen-specific immunoregulatory t (airt) cells
US20220380727A1 (en) 2019-11-08 2022-12-01 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
EP4103591A4 (en) 2020-02-13 2024-09-04 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
WO2022093884A1 (en) * 2020-10-27 2022-05-05 Pact Pharma, Inc. Compositions and methods for the treatment of cancer using next generation engineered t cell therapy
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

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1430124B1 (en) 2000-09-20 2013-11-06 EMD Millipore Corporation Artificial ubiquitous chromatin opening elements (ucoe)
US7507542B2 (en) 2001-05-08 2009-03-24 Ucb Sa Method for regulating immune function using the FOXP3 protein
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
EP2126054B1 (en) 2007-01-31 2016-07-06 Yeda Research And Development Company Limited 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
WO2008101272A1 (en) 2007-02-21 2008-08-28 Women's And Children's Health Research Institute Inc Method for obtaining treg-cells
US8158596B2 (en) 2007-05-11 2012-04-17 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
CA2717166A1 (en) 2008-03-03 2009-09-19 Converge Biotech Inc. Methods of modulating t cell-dependent immune responses
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
MX2015015638A (es) 2013-05-13 2016-10-28 Cellectis Metodos para diseñar celulas t altamente activas para inmunoterapia.
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
ES3063961T3 (en) 2014-02-14 2026-04-21 Cellectis Cells for immunotherapy engineered for targeting antigen present both on immune cells and pathological cells
EP3119897A1 (en) 2014-03-21 2017-01-25 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
CN107250373A (zh) 2015-01-12 2017-10-13 麻省理工学院 通过微流体递送实现的基因编辑
WO2016123578A1 (en) 2015-01-30 2016-08-04 The Regents Of The University Of California Protein delivery in primary hematopoietic cells
EP3294342A4 (en) * 2015-05-08 2018-11-07 President and Fellows of Harvard College Universal donor stem cells and related methods
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
WO2018035141A1 (en) 2016-08-16 2018-02-22 Bluebird Bio, Inc. Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use
WO2018073391A1 (en) * 2016-10-19 2018-04-26 Cellectis Targeted gene insertion for improved immune cells therapy
CA3041831A1 (en) 2016-10-27 2018-05-03 Intima Bioscience, Inc. Viral methods of making genetically modified cells
CN110139675B (zh) 2016-10-31 2023-09-29 西雅图儿童医院（Dba西雅图儿童研究所） 用具有工程化稳定的内源性foxp3基因表达的cd4t细胞治疗自身免疫疾病的方法
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
CN109797171A (zh) * 2017-05-08 2019-05-24 北京东方略细胞技术有限公司 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
US11713459B2 (en) * 2018-04-27 2023-08-01 Seattle Children's Hospital Expression of FOXP3 in edited CD34+ cells
CA3091491A1 (en) 2018-04-27 2019-10-31 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

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112851794A (zh) * 2021-02-04 2021-05-28 上海交通大学 一种基于cd271的新型抗原表位及其应用
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

Also Published As

Publication number Publication date
WO2019210042A1 (en) 2019-10-31
AU2019261438B2 (en) 2024-08-22
AU2024266824A1 (en) 2024-12-12
US20210054376A1 (en) 2021-02-25
JP2025013884A (ja) 2025-01-28
AU2019261438A1 (en) 2020-09-10
IL277039A (en) 2020-10-29
EP3784690A1 (en) 2021-03-03
SG11202007878UA (en) 2020-09-29
EP3784690A4 (en) 2022-01-19
US11713459B2 (en) 2023-08-01
CA3091688A1 (en) 2019-10-31
US20240117352A1 (en) 2024-04-11
JP7844582B2 (ja) 2026-04-13
JP7575950B2 (ja) 2024-10-31
JP2021521856A (ja) 2021-08-30

Similar Documents

Publication Publication Date Title
AU2019261438B2 (en) Expression of FOXP3 in edited CD34+ cells
AU2019257708B2 (en) Expression of human FOXP3 in gene edited T cells
KR102386029B1 (ko) 게놈 편집 면역 효과기 세포
AU2019279084B2 (en) Diverse antigen binding domains, novel platforms and other enhancements for cellular therapy
AU2017323629B9 (en) PD-1 homing endonuclease variants, compositions, and methods of use
AU2019375975B2 (en) Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons
KR20210149060A (ko) Tn7-유사 트랜스포존을 사용한 rna-유도된 dna 통합
CN108753824B (zh) 用于治疗视网膜营养不良的病毒载体
AU2016343979A1 (en) Delivery of central nervous system targeting polynucleotides
KR20210005179A (ko) 헤모글로빈-관련 돌연변이를 편집하기 위한 상동성-지정 복구 주형 설계 및 전달
CN108431225A (zh) 细胞基因组的诱导型修饰
CN112041436A (zh) 雷帕霉素抗性细胞
KR102628872B1 (ko) 세포의 증식을 제어하기 위해 세포 분열 좌위를 사용하기 위한 도구 및 방법
KR20210151785A (ko) 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
CN108136048A (zh) 左旋多巴的系统合成和调节
CN115768890A (zh) 通过分子和物理启动对t细胞免疫疗法的热控制
AU2018254529B2 (en) Therapeutic genome editing in Wiskott-Aldrich syndrome and X-linked thrombocytopenia
TW202317753A (zh) 經武裝之嵌合受體及其使用方法
KR20230069157A (ko) GJB2를 코딩하는 재조합 아데노 연관 바이러스 (rAAV) 및 그의 용도
AU2023412590A1 (en) Fusion and use thereof
HK40044016A (en) Expression of foxp3 in edited cd34+ cells
CN116997657A (zh) 编码GJB2的重组腺相关病毒(rAAV)及其用途
HK40041692A (en) Expression of human foxp3 in gene edited t cells
RU2812852C2 (ru) Невирусные днк-векторы и варианты их применения для экспрессии терапевтического средства на основе фактора viii (fviii)
NL2027815B1 (en) Genomic integration

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40044016

Country of ref document: HK