HK1259151A1 - 用於在造血干细胞中基因编辑的方法和组合物 - Google Patents

用於在造血干细胞中基因编辑的方法和组合物

Info

Publication number
HK1259151A1
HK1259151A1 HK19101177.2A HK19101177A HK1259151A1 HK 1259151 A1 HK1259151 A1 HK 1259151A1 HK 19101177 A HK19101177 A HK 19101177A HK 1259151 A1 HK1259151 A1 HK 1259151A1
Authority
HK
Hong Kong
Prior art keywords
hematopoietic stem
methods
compositions
stem cells
gene editing
Prior art date
Application number
HK19101177.2A
Other languages
English (en)
Chinese (zh)
Inventor
Saar GILL
Miriam KIM
Original Assignee
The Trustees Of The University Of Pennsylvania
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 The Trustees Of The University Of Pennsylvania filed Critical The Trustees Of The University Of Pennsylvania
Publication of HK1259151A1 publication Critical patent/HK1259151A1/zh

Links

Classifications

    • 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
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem 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
    • A61K48/005—Medicinal 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
    • 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
    • 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
    • A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31—Chimeric antigen receptors [CAR]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
    • A61K40/421—Immunoglobulin superfamily
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/05—Immunological preparations stimulating the reticulo-endothelial system, e.g. against cancer
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • 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]

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)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Hematology (AREA)
  • Developmental Biology & Embryology (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)
HK19101177.2A 2015-11-04 2016-11-03 用於在造血干细胞中基因编辑的方法和组合物 HK1259151A1 (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
HK1259151A1 true HK1259151A1 (zh) 2019-11-29

Family

ID=58662736

Family Applications (1)

Application Number Title Priority Date Filing Date
HK19101177.2A HK1259151A1 (zh) 2015-11-04 2016-11-03 用於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 (cg-RX-API-DMAC7.html)
EP (1) EP3370741B1 (cg-RX-API-DMAC7.html)
JP (3) JP6866385B2 (cg-RX-API-DMAC7.html)
KR (1) KR20180073679A (cg-RX-API-DMAC7.html)
CN (1) CN108463227A (cg-RX-API-DMAC7.html)
AU (2) AU2016349280B2 (cg-RX-API-DMAC7.html)
BR (1) BR112018009098A8 (cg-RX-API-DMAC7.html)
CA (1) CA3004053A1 (cg-RX-API-DMAC7.html)
CL (1) CL2018001198A1 (cg-RX-API-DMAC7.html)
EA (1) EA201891092A1 (cg-RX-API-DMAC7.html)
HK (1) HK1259151A1 (cg-RX-API-DMAC7.html)
IL (1) IL259084A (cg-RX-API-DMAC7.html)
MX (1) MX2018005612A (cg-RX-API-DMAC7.html)
MY (1) MY185961A (cg-RX-API-DMAC7.html)
PH (1) PH12018500965A1 (cg-RX-API-DMAC7.html)
SG (2) SG10202107602XA (cg-RX-API-DMAC7.html)
WO (1) WO2017079400A1 (cg-RX-API-DMAC7.html)

Families Citing this family (58)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2853829C (en) 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9163284B2 (en) 2013-08-09 2015-10-20 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
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
EP3177718B1 (en) 2014-07-30 2022-03-16 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
CN108290939B (zh) 2015-10-16 2023-01-13 纽约市哥伦比亚大学理事会 用于抑制谱系特异性抗原的组合物和方法
SG11201803173VA (en) 2015-10-23 2018-05-30 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
AU2017221456B2 (en) * 2016-02-19 2024-04-04 The Regents Of The University Of California Short hairpin RNA (shRNA734) and use of same to positively select and eliminate genetically modified cells
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
CA3033327A1 (en) 2016-08-09 2018-02-15 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
WO2018071868A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
HRP20240842T1 (hr) * 2016-11-02 2024-10-11 Universität Basel Imunološki uočljive varijante stanične površine za upotrebu u staničnoj terapiji
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
US11975029B2 (en) * 2017-02-28 2024-05-07 Vor Biopharma Inc. Compositions and methods for inhibition of lineage specific proteins
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
JP2020510439A (ja) 2017-03-10 2020-04-09 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 シトシンからグアニンへの塩基編集因子
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
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
AU2018352592C1 (en) 2017-10-16 2025-09-25 Beam Therapeutics, Inc. Uses of adenosine base editors
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
WO2019222545A1 (en) 2018-05-16 2019-11-21 Synthego Corporation Methods and systems for guide rna design and use
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
JP7716976B2 (ja) * 2018-09-19 2025-08-01 フジフィルム セルラー ダイナミクス，インコーポレイテッド キメラ抗原受容体改変免疫細胞の活性化及び拡大培養のためのプロテインｌ
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
EP3873205A4 (en) * 2018-10-31 2022-05-11 Humanigen, Inc. MATERIALS AND METHODS FOR TREATMENT OF CANCER
CN109852627B (zh) * 2018-12-26 2022-07-05 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
EP3911371A4 (en) * 2019-01-18 2023-04-26 University of Southern California METHODS AND COMPOSITIONS FOR IMPROVING THE SAFETY AND EFFICACY OF CELL THERAPY
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US20220202900A1 (en) * 2019-02-22 2022-06-30 The Trustees Of The University Of Pennsylvania 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
WO2020181101A1 (en) 2019-03-07 2020-09-10 The Regents Of The University Of California Crispr-cas effector polypeptides and methods of use thereof
GB2601617B (en) 2019-03-19 2024-02-21 Broad Inst Inc Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN114729367A (zh) * 2019-08-28 2022-07-08 Vor生物制药股份有限公司 用于cll1修饰的组合物和方法
CN114787352A (zh) * 2019-08-28 2022-07-22 Vor生物制药股份有限公司 用于cd123修饰的组合物和方法
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
AU2020361683A1 (en) 2019-10-11 2022-05-19 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
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
KR20230126707A (ko) * 2020-12-31 2023-08-30 보르 바이오파마 인크. Cd34 유전자 변형을 위한 조성물 및 방법
CN115443333B (zh) * 2021-04-27 2025-04-25 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
KR20240004702A (ko) * 2021-04-28 2024-01-11 에자이 알앤드디 매니지먼트 가부시키가이샤 안티센스 올리고뉴클레오티드 및 신경퇴행성 장애의 치료를 위한 이들의 용도
EP4430186A1 (en) * 2021-11-09 2024-09-18 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
CA3247767A1 (en) * 2022-01-11 2023-07-20 Actinium Pharmaceuticals, Inc. METHODS FOR THE TREATMENT OF CD33 POSITIVE MALIGNANT HEMATIC DISEASE
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

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2010515464A (ja) * 2007-01-11 2010-05-13 イエール・ユニバーシテイ Ｈｉｖ細胞表面受容体の標的化不活性化のための組成物および方法
CA2886684C (en) * 2012-10-10 2023-09-19 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
EP2958942B1 (en) * 2013-02-20 2020-06-03 Novartis AG Effective targeting of primary human leukemia using anti-cd123 chimeric antigen receptor engineered t cells
JP6608807B2 (ja) * 2013-05-29 2019-11-20 セレクティス Ｒｎａガイドｃａｓヌクレアーゼ系を用いることによって免疫療法のためにｔ細胞を操作するための方法
ES2876263T3 (es) * 2014-04-07 2021-11-12 Novartis Ag Tratamiento del cáncer usando receptor de antígeno quimérico anti-cd19
CN108290939B (zh) * 2015-10-16 2023-01-13 纽约市哥伦比亚大学理事会 用于抑制谱系特异性抗原的组合物和方法
KR20180073679A (ko) * 2015-11-04 2018-07-02 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 조혈 줄기 세포에서의 유전자 편집을 위한 방법 및 조성물

Also Published As

Publication number Publication date
BR112018009098A2 (pt) 2018-11-06
JP7378439B2 (ja) 2023-11-13
KR20180073679A (ko) 2018-07-02
EA201891092A1 (ru) 2018-10-31
US20200281975A1 (en) 2020-09-10
EP3370741A1 (en) 2018-09-12
EP3370741B1 (en) 2025-10-08
AU2016349280B2 (en) 2023-10-05
EP3370741A4 (en) 2019-04-24
SG11201803701YA (en) 2018-06-28
JP2018533625A (ja) 2018-11-15
AU2024200000A1 (en) 2024-03-21
IL259084A (en) 2018-06-28
EP3370741C0 (en) 2025-10-08
US10548922B2 (en) 2020-02-04
BR112018009098A8 (pt) 2019-02-26
WO2017079400A1 (en) 2017-05-11
MY185961A (en) 2021-06-14
CN108463227A (zh) 2018-08-28
CL2018001198A1 (es) 2018-09-21
MX2018005612A (es) 2018-11-09
US20240066064A1 (en) 2024-02-29
SG10202107602XA (en) 2021-08-30
US11771719B2 (en) 2023-10-03
JP2023115069A (ja) 2023-08-18
CA3004053A1 (en) 2017-05-11
PH12018500965A1 (en) 2018-11-12
AU2016349280A1 (en) 2018-05-24
JP2021106595A (ja) 2021-07-29
JP6866385B2 (ja) 2021-04-28
US20180250339A1 (en) 2018-09-06

Similar Documents

Publication Publication Date Title
HK1259151A1 (zh) 用於在造血干细胞中基因编辑的方法和组合物
EP4219725A3 (en) Altering gene expression in modified t cells and uses thereof
MX2020006117A (es) Integracion dirigida de acidos nucleicos.
EP3593812A3 (en) Treatment of cancer using chimeric antigen receptor
EA202091566A1 (ru) Содержащая нуклеиновую кислоту липидная наночастица и ее применение
HK1243333A1 (zh) 用於修饰的t细胞的方法和组合物
PH12018502400A1 (en) Anti-tim-3 antibodies and methods of use thereof
PH12019500709A1 (en) Anti-lag-3 antibodies and methods of use thereof
EP3950941A3 (en) Dnase polypeptide variants
MX383868B (es) Anticuerpos contra cd73 y sus usos.
MX2019006045A (es) Proteinas triespecificas dirigidas a psma y metodos de uso.
MX2022003116A (es) Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
MY200337A (en) Nucleic acid molecules encoding chimeric antigen receptors comprising a cd20 binding domain
WO2016014565A3 (en) Treatment of cancer using humanized anti-bcma chimeric antigen receptor
EA202090931A3 (ru) Композиции и способы перепрограммирования tcr с помощью гибридных белков
MY189028A (en) Anti-cd123 chimeric antigen receptor (car) for use in cancer treatment
PH12019500571A1 (en) Anti-pd-1 antibodies
EP3799878A3 (en) Repeat variable diresidues for targeting nucleotides
PH12020550483A1 (en) Anti-apoc3 antibodies and methods of use thereof
EP4446410A3 (en) Compositions comprising polypeptides having galactanase activity and polypeptides having beta-galactosidase activity
PH12014502182A1 (en) Methods and compositions for modulating notch activity
HK1247626A1 (zh) 一種由her2/neu(erbb2)受體蛋白衍生的369-377表位特異性的全人t細胞受體
HK1231768A1 (en) Treatment of cancer using a cll-1 chimeric antigen receptor
HK1231900A1 (en) Treatment of cancer using humanized anti-bcma chimeric antigen receptor
TW201612317A (en) Gene, composition and method for improving lignocelluloses hydrolyzation