CN108463227A - 在造血干细胞中基因编辑的方法和组合物 - Google Patents

在造血干细胞中基因编辑的方法和组合物 Download PDF

Info

Publication number
CN108463227A
CN108463227A CN201680076538.8A CN201680076538A CN108463227A CN 108463227 A CN108463227 A CN 108463227A CN 201680076538 A CN201680076538 A CN 201680076538A CN 108463227 A CN108463227 A CN 108463227A
Authority
CN
China
Prior art keywords
cell
cells
method described
endogenous gene
modification
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
CN201680076538.8A
Other languages
English (en)
Chinese (zh)
Inventor
S·吉尔
M·吉姆
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 Pennsylvania Penn
Original Assignee
University of Pennsylvania Penn
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 Pennsylvania Penn filed Critical University of Pennsylvania Penn
Publication of CN108463227A publication Critical patent/CN108463227A/zh
Pending legal-status Critical Current

Links

Classifications

    • 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
    • A61K48/005Medicinal 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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • 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
    • A61K35/17Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • 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
    • A61K35/28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/10Cellular immunotherapy characterised by the cell type used
    • A61K40/11T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/30Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31Chimeric antigen receptors [CAR]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/40Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41Vertebrate antigens
    • A61K40/42Cancer antigens
    • A61K40/4202Receptors, cell surface antigens or cell surface determinants
    • A61K40/421Immunoglobulin superfamily
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/05Immunological preparations stimulating the reticulo-endothelial system, e.g. against cancer
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • A61P35/02Antineoplastic agents specific for leukemia
    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • 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]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Oncology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CN201680076538.8A 2015-11-04 2016-11-03 在造血干细胞中基因编辑的方法和组合物 Pending CN108463227A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562250561P 2015-11-04 2015-11-04
US62/250,561 2015-11-04
PCT/US2016/060273 WO2017079400A1 (en) 2015-11-04 2016-11-03 Methods and compositions for gene editing in hematopoietic stem cells

Publications (1)

Publication Number Publication Date
CN108463227A true CN108463227A (zh) 2018-08-28

Family

ID=58662736

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201680076538.8A Pending CN108463227A (zh) 2015-11-04 2016-11-03 在造血干细胞中基因编辑的方法和组合物

Country Status (17)

Country Link
US (3) US10548922B2 (enExample)
EP (1) EP3370741B1 (enExample)
JP (3) JP6866385B2 (enExample)
KR (1) KR20180073679A (enExample)
CN (1) CN108463227A (enExample)
AU (2) AU2016349280B2 (enExample)
BR (1) BR112018009098A8 (enExample)
CA (1) CA3004053A1 (enExample)
CL (1) CL2018001198A1 (enExample)
EA (1) EA201891092A1 (enExample)
HK (1) HK1259151A1 (enExample)
IL (1) IL259084A (enExample)
MX (1) MX2018005612A (enExample)
MY (1) MY185961A (enExample)
PH (1) PH12018500965A1 (enExample)
SG (2) SG11201803701YA (enExample)
WO (1) WO2017079400A1 (enExample)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109852627A (zh) * 2018-12-26 2019-06-07 中国人民解放军军事科学院军事医学研究院 用于CRISPRi系统的质粒、其构建方法及其在使鼠疫菌目的基因定向沉默中的应用
CN110662554A (zh) * 2017-02-28 2020-01-07 Vor生物制药股份有限公司 用于抑制谱系特异性蛋白质的组合物和方法
CN113194715A (zh) * 2018-10-31 2021-07-30 赫曼尼根公司 用于治疗癌症的材料和方法
WO2022228471A1 (zh) * 2021-04-27 2022-11-03 上海驯鹿生物技术有限公司 一种基因编辑的造血干细胞及其与car-t细胞的联合应用

Families Citing this family (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6261500B2 (ja) 2011-07-22 2018-01-17 プレジデント アンド フェローズ オブ ハーバード カレッジ ヌクレアーゼ切断特異性の評価および改善
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
EP3025719B1 (en) 2014-11-26 2018-09-26 Miltenyi Biotec GmbH Combination immunotherapy of antigen-recognizing receptors and hematopoietic cells for the treatment of diseases
JP7017506B2 (ja) 2015-10-16 2022-02-08 ザ・トラスティーズ・オブ・コロンビア・ユニバーシティ・イン・ザ・シティ・オブ・ニューヨーク 系統特異的抗原の阻害のための組成物および方法
EP3365356B1 (en) 2015-10-23 2023-06-28 President and Fellows of Harvard College Nucleobase editors and uses thereof
KR20180073679A (ko) * 2015-11-04 2018-07-02 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 조혈 줄기 세포에서의 유전자 편집을 위한 방법 및 조성물
CN108495640B (zh) * 2016-02-19 2022-11-04 加利福尼亚大学董事会 短发夹rna(shrna734)及其用于阳性选择和消除遗传修饰的细胞的用途
JP7231935B2 (ja) 2016-08-03 2023-03-08 プレジデント アンド フェローズ オブ ハーバード カレッジ アデノシン核酸塩基編集因子およびそれらの使用
EP3497214B1 (en) 2016-08-09 2023-06-28 President and Fellows of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
CN110191948A (zh) * 2016-11-02 2019-08-30 巴塞尔大学 用于细胞疗法的免疫可识别的细胞表面变体
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
CN112654710A (zh) 2018-05-16 2021-04-13 辛瑟高公司 用于指导rna设计和使用的方法和系统
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
IL260445B (en) * 2018-07-05 2019-08-29 Lempo Therapeutics Ltd Methods and compositions for modulating myeloperoxidase (mpo) expression
CN113423725A (zh) 2018-08-28 2021-09-21 Vor生物制药股份有限公司 遗传工程化造血干细胞及其用途
US20220031744A1 (en) * 2018-09-19 2022-02-03 FUJIFILM Cellular Dynamics, Inc. Protein l for activation and expansion of chimeric antigen receptor-modified immune cells
US12331320B2 (en) 2018-10-10 2025-06-17 The Research Foundation For The State University Of New York Genome edited cancer cell vaccines
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
WO2020150702A1 (en) * 2019-01-18 2020-07-23 University Of Southern California Methods and compositions to improve the safety and efficacy of cellular therapies
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020172638A1 (en) * 2019-02-22 2020-08-27 The Trustees Of The University Of Pennsylvania Crispr/cas9 knock-out of cd33 in human hematopoietic stem/ progenitor cells for allogenic transplantation
MX2021010559A (es) 2019-03-07 2021-12-15 Univ California Polipéptidos efectores de crispr-cas y métodos de uso de estos.
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
KR20220047381A (ko) * 2019-08-28 2022-04-15 보르 바이오파마 인크. Cd123 변형을 위한 조성물 및 방법
CA3151638A1 (en) * 2019-08-28 2021-03-04 Vor Biopharma Inc. Compositions and methods for cll1 modification
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CA3154280A1 (en) 2019-10-11 2021-04-15 The Trustees Of The University Of Pennsylvania Compositions and methods for targeting cd13 and tim-3 with car t cells to treat acute myeloid leukemia
KR20210109971A (ko) 2020-02-28 2021-09-07 현대자동차주식회사 리튬이온 전지 음극 제조방법 및 이에 의해 제조된 리튬이온 전지 음극을 포함하는 리튬이온 전지
MX2022014008A (es) 2020-05-08 2023-02-09 Broad Inst Inc Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo.
KR20230126707A (ko) * 2020-12-31 2023-08-30 보르 바이오파마 인크. Cd34 유전자 변형을 위한 조성물 및 방법
JP2024518780A (ja) * 2021-04-28 2024-05-02 エーザイ・アール・アンド・ディー・マネジメント株式会社 神経変性障害の治療用アンチセンスオリゴヌクレオチド及びその使用
CA3236152A1 (en) * 2021-11-09 2023-05-19 Vor Biopharma Inc. Compositions and methods for erm2 modification
WO2023091954A2 (en) 2021-11-19 2023-05-25 The Trustees Of The University Of Pennsylvania Engineered pan-leukocyte antigen cd45 to facilitate car t cell therapy
US20250073274A1 (en) * 2022-01-11 2025-03-06 Actinium Pharmaceuticals, Inc. Methods for treating cd33-positive hematological malignancies
WO2025030010A1 (en) 2023-08-01 2025-02-06 Vor Biopharma Inc. Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof
WO2025059062A1 (en) * 2023-09-11 2025-03-20 The Trustees Of The University Of Pennsylvania Genetic engineering of human hematopoietic stem/progenitor cells (hspcs) for locus-specific expression of therapeutic proteins

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100172882A1 (en) * 2007-01-11 2010-07-08 Glazer Peter M Compositions and methods for targeted inactivation of hiv cell surface receptors
WO2014059173A2 (en) * 2012-10-10 2014-04-17 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
US20140322212A1 (en) * 2013-02-20 2014-10-30 Jennifer Brogdon Effective targeting of primary human leukemia using anti-cd123 chimeric antigen receptor engineered t cells
WO2014191128A1 (en) * 2013-05-29 2014-12-04 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
US20150283178A1 (en) * 2014-04-07 2015-10-08 Carl H. June Treatment of cancer using anti-cd19 chimeric antigen receptor

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7017506B2 (ja) * 2015-10-16 2022-02-08 ザ・トラスティーズ・オブ・コロンビア・ユニバーシティ・イン・ザ・シティ・オブ・ニューヨーク 系統特異的抗原の阻害のための組成物および方法
KR20180073679A (ko) * 2015-11-04 2018-07-02 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 조혈 줄기 세포에서의 유전자 편집을 위한 방법 및 조성물

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100172882A1 (en) * 2007-01-11 2010-07-08 Glazer Peter M Compositions and methods for targeted inactivation of hiv cell surface receptors
WO2014059173A2 (en) * 2012-10-10 2014-04-17 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
US20140322212A1 (en) * 2013-02-20 2014-10-30 Jennifer Brogdon Effective targeting of primary human leukemia using anti-cd123 chimeric antigen receptor engineered t cells
WO2014191128A1 (en) * 2013-05-29 2014-12-04 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
US20150283178A1 (en) * 2014-04-07 2015-10-08 Carl H. June Treatment of cancer using anti-cd19 chimeric antigen receptor

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
DANIEL J. PEARCE等: "Implications of the Expression of Myeloid Markers on Normal and Leukemic Stem Cells", 《CELL CYCLE》 *
JOHN G DOENCH等: "Rational design of highly active sgRNAs for CRISPR-Cas9-mediated gene inactivation", 《NATURE BIOTECHNOLOGY》 *
LISA M OTT DE BRUIN 等: "Novel Genome-Editing Tools to Model and Correct Primary Immunodeficiencies", 《FRONTIERS IN IMMUNOLOGY》 *
MICHAEL J. ROBERTSON等: "Human Bone Marrow Depleted of CD33-Positive Cells Mediates Delayed but Durable Reconstitution of Hematopoiesis: Clinical Trial of MY9 Monoclonal Antibody-Purged Autografts for the Treatment of Acute Myeloid Leukemia", 《BLOOD》 *
PANKAJ K. MANDAL 等: "Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9", 《CELL STEM CELL》 *
VIRNA MARIN 等: "Cytokine-induced killer cells for cell therapy of acute myeloid leukemia: improvement of their immune activity by expression of CD33-specific chimeric receptors", 《HAEMATOLOGICA》 *
XIAO-LING LIU等: "Differential gene expression in human hematopoietic stem cells specified toward erythroid, megakaryocytic, and granulocytic lineage", 《JOURNAL OF LEUKOCYTE BIOLOGY》 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110662554A (zh) * 2017-02-28 2020-01-07 Vor生物制药股份有限公司 用于抑制谱系特异性蛋白质的组合物和方法
CN113194715A (zh) * 2018-10-31 2021-07-30 赫曼尼根公司 用于治疗癌症的材料和方法
CN113194715B (zh) * 2018-10-31 2024-03-19 赫曼尼根公司 用于治疗癌症的材料和方法
CN109852627A (zh) * 2018-12-26 2019-06-07 中国人民解放军军事科学院军事医学研究院 用于CRISPRi系统的质粒、其构建方法及其在使鼠疫菌目的基因定向沉默中的应用
WO2022228471A1 (zh) * 2021-04-27 2022-11-03 上海驯鹿生物技术有限公司 一种基因编辑的造血干细胞及其与car-t细胞的联合应用

Also Published As

Publication number Publication date
PH12018500965A1 (en) 2018-11-12
US20180250339A1 (en) 2018-09-06
AU2016349280B2 (en) 2023-10-05
EP3370741A1 (en) 2018-09-12
HK1259151A1 (zh) 2019-11-29
SG11201803701YA (en) 2018-06-28
AU2016349280A1 (en) 2018-05-24
EP3370741C0 (en) 2025-10-08
BR112018009098A2 (pt) 2018-11-06
BR112018009098A8 (pt) 2019-02-26
SG10202107602XA (en) 2021-08-30
EP3370741A4 (en) 2019-04-24
JP7378439B2 (ja) 2023-11-13
AU2024200000A1 (en) 2024-03-21
EA201891092A1 (ru) 2018-10-31
JP2023115069A (ja) 2023-08-18
EP3370741B1 (en) 2025-10-08
JP6866385B2 (ja) 2021-04-28
US20240066064A1 (en) 2024-02-29
CL2018001198A1 (es) 2018-09-21
CA3004053A1 (en) 2017-05-11
MX2018005612A (es) 2018-11-09
WO2017079400A1 (en) 2017-05-11
JP2018533625A (ja) 2018-11-15
IL259084A (en) 2018-06-28
MY185961A (en) 2021-06-14
US10548922B2 (en) 2020-02-04
US11771719B2 (en) 2023-10-03
US20200281975A1 (en) 2020-09-10
KR20180073679A (ko) 2018-07-02
JP2021106595A (ja) 2021-07-29

Similar Documents

Publication Publication Date Title
JP7378439B2 (ja) 造血幹細胞における遺伝子編集のための方法および組成物
CN107206024B (zh) 改变cart细胞中的基因表达及其用途
JP2025061285A (ja) Crispr/cpf1を用いる、t細胞における遺伝子編集のための組成物および方法
JP2017533706A (ja) 改変t細胞に関する方法および組成物
US20220202900A1 (en) Compositions and methods for crispr/cas9 knock-out of cd33 in human hematopoietic stem / progenitor cells for allogenic transplantation in patients with relapsed - refractory acute myeloid leukemia
US20220041984A1 (en) Mobilized peripheral blood as a source of modified immune cells
HK40112241A (en) Altering gene expression in car-t cells and uses thereof
HK1243332B (en) Altering gene expression in modified t cells and uses thereof

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