IL277039A - Expression of FOXP3 in edited CD34 cells - Google Patents

Expression of FOXP3 in edited CD34 cells

Info

Publication number
IL277039A
IL277039A IL277039A IL27703920A IL277039A IL 277039 A IL277039 A IL 277039A IL 277039 A IL277039 A IL 277039A IL 27703920 A IL27703920 A IL 27703920A IL 277039 A IL277039 A IL 277039A
Authority
IL
Israel
Prior art keywords
foxp3
edited
expression
cells
Prior art date
Application number
IL277039A
Other languages
English (en)
Hebrew (he)
Inventor
Andrew M Scharenberg
David J Rawlings
Karen Sommer
Yuchi Chiang Honaker
Iram F Khan
Swati Singh
Original Assignee
Seattle Childrens Hospital Dba Seattle Childrens Res Inst
Andrew M Scharenberg
David J Rawlings
Karen Sommer
Yuchi Chiang Honaker
Iram F Khan
Swati Singh
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 Dba Seattle Childrens Res Inst, Andrew M Scharenberg, David J Rawlings, Karen Sommer, Yuchi Chiang Honaker, Iram F Khan, Swati Singh filed Critical Seattle Childrens Hospital Dba Seattle Childrens Res Inst
Publication of IL277039A publication Critical patent/IL277039A/en

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • 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
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    • 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
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    • 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
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    • 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
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2510/00—Genetically modified cells
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    • 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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (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)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Rheumatology (AREA)
  • Rehabilitation Therapy (AREA)
  • Diabetes (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)
IL277039A 2018-04-27 2020-08-31 Expression of FOXP3 in edited CD34 cells IL277039A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862663545P 2018-04-27 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
IL277039A true IL277039A (en) 2020-10-29

Family

ID=68294731

Family Applications (1)

Application Number Title Priority Date Filing Date
IL277039A IL277039A (en) 2018-04-27 2020-08-31 Expression of FOXP3 in edited CD34 cells

Country Status (9)

Country Link
US (2) US11713459B2 (enExample)
EP (1) EP3784690A4 (enExample)
JP (3) 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)

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JP7575949B2 (ja) 2018-04-27 2024-10-31 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 遺伝子編集ｔ細胞におけるヒトｆｏｘｐ３の発現
SG11202007878UA (en) * 2018-04-27 2020-09-29 Seattle Childrens Hospital Dba Seattle Childrens Res Inst Expression of foxp3 in edited cd34+ cells
AU2020209687A1 (en) * 2019-01-18 2021-07-29 Osaka University Composition for use in treating dystrophic epidermolysis bullosa
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EP3980035A4 (en) 2019-06-07 2023-08-23 The Board Of Trustees Of The Leland Stanford Junior University GENETICALLY MODIFIED CD4+ T LYMPHOCYTES FOR USE IN TREG-BASED IMMUNOTHERAPY
AU2020304057A1 (en) * 2019-06-27 2022-01-27 Benaroya Research Institute At Virginia Mason Artificial antigen-specific immunoregulatory t (airt) cells
CA3160113A1 (en) 2019-11-08 2021-05-14 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
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
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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
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JP7575949B2 (ja) 2018-04-27 2024-10-31 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 遺伝子編集ｔ細胞におけるヒトｆｏｘｐ３の発現
SG11202007878UA (en) * 2018-04-27 2020-09-29 Seattle Childrens Hospital Dba Seattle Childrens Res Inst Expression of foxp3 in edited cd34+ cells
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Publication number Publication date
JP2021521856A (ja) 2021-08-30
US11713459B2 (en) 2023-08-01
US20240117352A1 (en) 2024-04-11
JP2026108825A (ja) 2026-06-30
AU2019261438B2 (en) 2024-08-22
JP7575950B2 (ja) 2024-10-31
CA3091688A1 (en) 2019-10-31
JP2025013884A (ja) 2025-01-28
EP3784690A1 (en) 2021-03-03
EP3784690A4 (en) 2022-01-19
SG11202007878UA (en) 2020-09-29
AU2019261438A1 (en) 2020-09-10
AU2024266824A1 (en) 2024-12-12
US20210054376A1 (en) 2021-02-25
CN112218882A (zh) 2021-01-12
WO2019210042A1 (en) 2019-10-31
JP7844582B2 (ja) 2026-04-13

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