CA3091688A1 - Expression of foxp3 in edited cd34+ cells - Google Patents

Expression of foxp3 in edited cd34+ cells Download PDF

Info

Publication number
CA3091688A1
CA3091688A1 CA3091688A CA3091688A CA3091688A1 CA 3091688 A1 CA3091688 A1 CA 3091688A1 CA 3091688 A CA3091688 A CA 3091688A CA 3091688 A CA3091688 A CA 3091688A CA 3091688 A1 CA3091688 A1 CA 3091688A1
Authority
CA
Canada
Prior art keywords
sequence
cell
foxp3
nucleic acid
seq
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
CA3091688A
Other languages
English (en)
French (fr)
Inventor
David J. Rawlings
Iram F. KHAN
Yuchi Chiang HONAKER
Swati SINGH
Karen SOMMER
Andrew M. Scharenberg
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 CA3091688A1 publication Critical patent/CA3091688A1/en
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • 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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Developmental Biology & Embryology (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)
CA3091688A 2018-04-27 2019-04-25 Expression of foxp3 in edited cd34+ cells Pending CA3091688A1 (en)

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
CA3091688A1 true CA3091688A1 (en) 2019-10-31

Family

ID=68294731

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3091688A Pending CA3091688A1 (en) 2018-04-27 2019-04-25 Expression of foxp3 in edited cd34+ cells

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=)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3042179A1 (en) 2016-10-31 2018-05-03 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 endogenous foxp3 gene
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
AU2019261438B2 (en) * 2018-04-27 2024-08-22 Seattle Children's Hospital (dba Seattle Children's Research Institute) 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
JP2022533713A (ja) 2019-05-21 2022-07-25 サンガモ セラピューティクス， インコーポレイテッド 調節性ｔ細胞における制御された導入遺伝子発現
WO2020264039A1 (en) * 2019-06-27 2020-12-30 Seattle Children's Hospital (dba Seattle Children's Research Institute) Artificial antigen-specific immunoregulatory t (airt) cells
JP2023501388A (ja) 2019-11-08 2023-01-18 サンガモ セラピューティクス， インコーポレイテッド 操作された制御性ｔ細胞の生成
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
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
GB202113673D0 (en) 2021-09-24 2021-11-10 Reflection Therapeutics Ltd Targeted cell therapies
GB202113674D0 (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
KR100996016B1 (ko) 2000-09-20 2010-11-22 밀리포어 코포레이션 폴리뉴클레오티드
EP1967525B1 (en) 2001-05-08 2012-11-14 Darwin Molecular Corporation A method for regulating immune function in primates 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
US8846308B2 (en) 2007-06-08 2014-09-30 The Trustees Of The University Of Pennsylvania Method for identifying immune response modulators
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
TW200942246A (en) 2008-03-03 2009-10-16 Fabio Grassi Method 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
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
WO2014191128A1 (en) 2013-05-29 2014-12-04 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
WO2015121454A1 (en) 2014-02-14 2015-08-20 Cellectis Cells for immunotherapy engineered for targeting antigen present both on immune cells and pathological cells
AU2015233347A1 (en) 2014-03-21 2016-09-08 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
CN107921148A (zh) * 2015-05-08 2018-04-17 哈佛学院校长同事会 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
EP3497221A4 (en) 2016-08-10 2020-02-05 Duke University COMPOSITIONS, SYSTEMS AND METHODS FOR PROGRAMMING IMMUNE CELL FUNCTION BY TARGETED GENE REGULATION
MA45996A (fr) * 2016-08-16 2021-06-02 Bluebird Bio Inc Variants d'endonucléase de homing du récepteur alpha de l'il-10, compositions et méthodes d'utilisation associées
WO2018073391A1 (en) 2016-10-19 2018-04-26 Cellectis Targeted gene insertion for improved immune cells therapy
AU2017347854B2 (en) 2016-10-27 2022-12-08 Intima Bioscience, Inc. Viral methods of T cell therapy
CA3042179A1 (en) 2016-10-31 2018-05-03 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 endogenous foxp3 gene
CN110191955B (zh) 2016-12-13 2024-05-31 西雅图儿童医院（Dba西雅图儿童研究所） 在体外和体内对工程化的细胞中表达的化学物质诱导的信号传导复合物进行外源性药物激活的方法
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
WO2019040655A1 (en) 2017-08-22 2019-02-28 The Regents Of The University Of California LENTIVIRAL VECTORS EXPRESSING FOXP3 IN HEMATOPOIETIC STEM CELLS TO TREAT IMMUNITY DEFICIENCIES AND AUTOIMMUNE DISEASES
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
AU2019261438B2 (en) * 2018-04-27 2024-08-22 Seattle Children's Hospital (dba Seattle Children's Research Institute) Expression of FOXP3 in edited CD34+ cells
WO2019241549A1 (en) 2018-06-15 2019-12-19 A2 Biotherapeutics, Inc. Foxp3-expressing car-t regulatory cells

Also Published As

Publication number Publication date
SG11202007878UA (en) 2020-09-29
US11713459B2 (en) 2023-08-01
WO2019210042A1 (en) 2019-10-31
CN112218882A (zh) 2021-01-12
US20210054376A1 (en) 2021-02-25
AU2019261438B2 (en) 2024-08-22
JP2021521856A (ja) 2021-08-30
JP2025013884A (ja) 2025-01-28
IL277039A (en) 2020-10-29
JP7575950B2 (ja) 2024-10-31
EP3784690A4 (en) 2022-01-19
AU2019261438A1 (en) 2020-09-10
JP7844582B2 (ja) 2026-04-13
US20240117352A1 (en) 2024-04-11
EP3784690A1 (en) 2021-03-03
AU2024266824A1 (en) 2024-12-12

Similar Documents

Publication Publication Date Title
US20240117352A1 (en) Expression of foxp3 in edited cd34+ cells
JP7844581B2 (ja) 遺伝子編集ｔ細胞におけるヒトｆｏｘｐ３の発現
KR102728416B1 (ko) A형 혈우병을 위한 유전자 편집용 조성물 및 방법
JP7480062B2 (ja) ラパマイシン耐性細胞
KR102796744B1 (ko) 이식의 개선을 위한 crispr/cas-관련 방법 및 조성물
AU2016261358B2 (en) Optimized CRISPR/Cas9 systems and methods for gene editing in stem cells
CN112020558A (zh) 治疗血红蛋白病的系统和方法
CA2985615A1 (en) Crispr/cas-related methods and compositions for treating hiv infection and aids
EP3155098A1 (en) FACTOR VIII MUTATION REPAIR AND TOLERANCE INDUCTION AND RELATED CDNAs, COMPOSITIONS, METHODS AND SYSTEMS
US12214055B2 (en) Systems and methods for treating hyper-igm syndrome
US20240327862A1 (en) Methods of Treating Rheumatoid Arthritis Using RNA-Guided Genome Editing of HLA Gene
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
KR20220022110A (ko) 개선된 인자 viii 발현을 사용한, a형 혈우병을 위한 유전자 편집

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20240418

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 6TH ANNIV.) - STANDARD

Year of fee payment: 6

U00 Fee paid

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

Effective date: 20241227

U11 Full renewal or maintenance fee paid

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

Effective date: 20241227

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

Effective date: 20241227

D15 Examination report completed

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

Effective date: 20251002