CL2018001198A1 - Métodos y composiciones para edición génica en hemocitoblastos - Google Patents

Métodos y composiciones para edición génica en hemocitoblastos

Info

Publication number
CL2018001198A1
CL2018001198A1 CL2018001198A CL2018001198A CL2018001198A1 CL 2018001198 A1 CL2018001198 A1 CL 2018001198A1 CL 2018001198 A CL2018001198 A CL 2018001198A CL 2018001198 A CL2018001198 A CL 2018001198A CL 2018001198 A1 CL2018001198 A1 CL 2018001198A1
Authority
CL
Chile
Prior art keywords
compositions
modified
methods
hemocytoblasts
hemocytoblast
Prior art date
Application number
CL2018001198A
Other languages
English (en)
Spanish (es)
Inventor
Saar Gill
Miriam Kim
Original Assignee
Univ 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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of CL2018001198A1 publication Critical patent/CL2018001198A1/es

Links

Classifications

    • 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
    • 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
    • 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)
  • 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)
CL2018001198A 2015-11-04 2018-05-03 Métodos y composiciones para edición génica en hemocitoblastos CL2018001198A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562250561P 2015-11-04 2015-11-04

Publications (1)

Publication Number Publication Date
CL2018001198A1 true CL2018001198A1 (es) 2018-09-21

Family

ID=58662736

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2018001198A CL2018001198A1 (es) 2015-11-04 2018-05-03 Métodos y composiciones para edición génica en hemocitoblastos

Country Status (17)

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

Families Citing this family (58)

* 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
BR112019017751A2 (pt) * 2017-02-28 2020-04-07 Vor Biopharma, Inc. composições e métodos de inibição de proteínas de linhagem específica
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
CA3116412A1 (en) * 2018-10-31 2020-05-07 Humanigen, Inc. Materials and methods for treating 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
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
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
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

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
ES2824024T3 (es) * 2012-10-10 2021-05-11 Sangamo Therapeutics Inc Compuestos modificadores de células T y usos de los mismos
EP3744736A1 (en) * 2013-02-20 2020-12-02 Novartis AG Effective targeting of primary human leukemia using anti-cd123 chimeric antigen receptor engineered t cells
EP3309248B1 (en) * 2013-05-29 2021-06-09 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
SI3888674T1 (sl) * 2014-04-07 2024-08-30 Novartis Ag Zdravljenje raka z uporabo antigenskega himernega receptorja proti-CD19
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

Also Published As

Publication number Publication date
CN108463227A (zh) 2018-08-28
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
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
CL2018001198A1 (es) Métodos y composiciones para edición génica en hemocitoblastos
MX2024001192A (es) Anticuerpos anti gen 3 de activacion linfocitica (lag-3) y metodos para usarlos.
BR112019007288A2 (pt) proteína heterodimérica biespecífica, composições de ácido nucleico e de vetor de expressão, vetor de expressão, célula hospedeira, e, métodos para produzir proteína heterodimérica biespecífica e para tratar câncer em um paciente
MX2017001011A (es) Tratamiento de cancer de usando un receptor quimerico de antigeno anti-bcma.
BR112018070676A2 (pt) construtos de ligação a antígeno multiespecífico que têm como alvo agentes imunoterapêuticos
BR112017000939A2 (pt) tratamento de câncer usando um receptor antigênico quimérico de cll-1
ECSP17028310A (es) Composiciones y métodos para inhibir la expresión del gen HAO1 (hidroxiácido-oxidasa 1 (glicolato-oxidasa))
MX2023004169A (es) Alteracion de la expresion genetica en celulas t modificadas con el receptor de antigeno quimerico (cart) y usos de las mismas.
MX2018010733A (es) Celulas que expresan multiples moleculas del receptor de antigeno quimerico (car) y usos de las mismas.
MX2023000818A (es) Anticuerpos anti-antigeno 4 del linfocito t citotoxico (ctla-4) y metodos para uso de los mismos.
CO2017010190A2 (es) Terapias cd20, terapias cd22 y terapias de combinación con una célula que expresa un receptor quimérico de antígeno (car) de cd19
MX2017002205A (es) Receptor quimerico de antigeno (car) anti-cd123 para uso en el tratamiento de cancer.
BR112018005937A2 (pt) conjugados de oligonucleotídeo de afinidade e usos destes
EA202090931A3 (ru) Композиции и способы перепрограммирования tcr с помощью гибридных белков
PE20170772A1 (es) Anticuerpo monoclonal anti-ctla4 o su fragmento de union a antigeno, una composicion farmaceutica y uso
CO2019003951A2 (es) Proteínas de unión a cd123 y composiciones y métodos relacionados
BR112015021819A2 (pt) Tratamento de câncer utilizando receptor quimérico de antígeno humanizado anti-cd19
MX2015010836A (es) Tratamiento de cancer utilizando un receptor quimerico de antigeno anti egfrviii humanizado.
MX2023002858A (es) Prediccion de epitopos de celulas t utiles para vacunacion.
UY36404A (es) ANTICUERPOS MONOCLONALES (Ab) COMO DETECTORES DE CD73 E INHIBIDORES DE SU ACTIVIDAD ENZIMÁTICA, Y COMPOSICIONES QUE LOS CONTIENEN
AR102879A1 (es) Métodos para el tratamiento de tumores cerebrales
MX2017014908A (es) Proteinas de union triespecificas y metodos de uso.
MX382997B (es) Composiciones y metodos para mejorar la expresion genetica de pklr.
AR118751A2 (es) Tratamiento de cáncer utilizando receptor de antígeno quimérico anti-cd19
EA201990927A1 (ru) Антитела против lag-3 и способы их применения