JP2023552816A - 細胞内のｍｈｃクラスｉｉを低減する組成物及び方法 - Google Patents

細胞内のｍｈｃクラスｉｉを低減する組成物及び方法 Download PDF

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JP2023552816A
JP2023552816A JP2023534899A JP2023534899A JP2023552816A JP 2023552816 A JP2023552816 A JP 2023552816A JP 2023534899 A JP2023534899 A JP 2023534899A JP 2023534899 A JP2023534899 A JP 2023534899A JP 2023552816 A JP2023552816 A JP 2023552816A
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ciita
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スリダー，スリジャニ
ザン，ヨン
フレデリック ハリントン，ウィリアム
ゴエル，サービ
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インテリア セラピューティクス，インコーポレイテッド
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Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4544058A1 (en) * 2022-06-23 2025-04-30 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of ciita expression
US20240189355A1 (en) * 2022-11-14 2024-06-13 WUGEN, Inc. Hypoimmunogenic cells having targeted modifications in mhc class-i genes and methods of use
CN118922551A (zh) * 2023-01-30 2024-11-08 南京北恒生物科技有限公司 用于CRISPR-Cas基因编辑系统的方法及组合物
KR102740042B1 (ko) 2023-07-12 2024-12-06 ㈜바이오솔빅스 인간 전분화능 줄기세포로 유래된 심장 오가노이드 제조방법, 효능 및 독성 평가 기술
WO2025038646A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Cd70 car-t compositions and methods for cell-based therapy
WO2025038637A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
WO2025038642A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying cd70
IL326191A (en) 2023-08-14 2026-03-01 Intellia Therapeutics Inc Compositions and methods for genetic modification of transforming growth factor beta receptor type 2 (tgfβr2)
WO2025049432A1 (en) * 2023-08-28 2025-03-06 Intellia Therapeutics, Inc. Methods for editing hla-a in cells pre-screened for the absence of one or both alleles of hla-h*01
WO2025049481A1 (en) * 2023-08-28 2025-03-06 Intellia Therapeutics, Inc. Methods of editing an hla-a gene in vitro
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025255308A1 (en) 2024-06-07 2025-12-11 Intellia Therapeutics, Inc. Cd8 co-receptor chimeric polypeptides in tcr cell therapy

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
JP3131222B2 (ja) 1991-12-24 2001-01-31 アイシス・ファーマシューティカルス・インコーポレーテッド ギャップを有する２′修飾オリゴヌクレオチド
JPH10500310A (ja) 1994-05-19 1998-01-13 ダコ アクティーゼルスカブ 淋菌及びトラコーマ クラミジアの検出のためのｐｎａ プローブ
CN103668470B (zh) 2012-09-12 2015-07-29 上海斯丹赛生物技术有限公司 一种dna文库及构建转录激活子样效应因子核酸酶质粒的方法
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
DK2931891T3 (da) 2012-12-17 2019-08-19 Harvard College Rna-styret modificering af menneskelige genomer
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
CN106794141B (zh) 2014-07-16 2021-05-28 诺华股份有限公司 将核酸包封在脂质纳米粒主体中的方法
EP2990416B1 (en) 2014-08-29 2018-06-20 GEMoaB Monoclonals GmbH Universal chimeric antigen receptor expressing immune cells for targeting of diverse multiple antigens and method of manufacturing the same and use of the same for treatment of cancer, infections and autoimmune disorders
KR20230156800A (ko) 2015-03-03 2023-11-14 더 제너럴 하스피탈 코포레이션 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제
WO2017093969A1 (en) * 2015-12-04 2017-06-08 Novartis Ag Compositions and methods for immunooncology
LT3436077T (lt) 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
WO2018073393A2 (en) 2016-10-19 2018-04-26 Cellectis Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy
SG10202106058WA (en) 2016-12-08 2021-07-29 Intellia Therapeutics Inc Modified guide rnas
EP4029943A1 (en) 2017-05-08 2022-07-20 Precision Biosciences, Inc. Nucleic acid molecules encoding an engineered antigen receptor and an inhibitory nucleic acid molecule and methods of use thereof
MX2019013514A (es) * 2017-05-12 2020-01-20 Crispr Therapeutics Ag Materiales y metodos para modificar celulas por ingenieria genetica y usos de los mismos en inmunooncologia.
US20230140670A1 (en) 2017-09-29 2023-05-04 Intellia Therapeutics, Inc. Formulations
EP3724326A1 (en) * 2017-12-11 2020-10-21 Editas Medicine, Inc. Cpf1-related methods and compositions for gene editing
WO2019147805A2 (en) 2018-01-26 2019-08-01 The Board Of Trustees Of The Leland Stanford Junior University Regulatory t cells targeted with chimeric antigen receptors
US11447769B2 (en) 2018-03-27 2022-09-20 The Trustees Of The University Of Pennsylvania Modified immune cells having enhanced function and methods for screening for same
WO2019237035A1 (en) * 2018-06-08 2019-12-12 Intellia Therapeutics, Inc. Compositions and methods for immunooncology
AU2019282824C1 (en) 2018-06-08 2026-04-23 Intellia Therapeutics, Inc. Modified guide RNAS for gene editing
EP3581200A1 (en) 2018-06-13 2019-12-18 GEMoaB Monoclonals GmbH Reversed universal chimeric antigen receptor expressing immune cells for targeting of diverse multiple antigens and method of manufacturing the same and use of the same for treatment of cancer, infections and autoimmune disorders
IL282267B2 (en) 2018-10-15 2026-03-01 Univ Massachusetts Programmed DNA editing by chimeric deaminase-nme2cas9 proteins
CN113227374A (zh) 2018-10-16 2021-08-06 因特利亚治疗公司 用于免疫疗法的组合物和方法
WO2020092057A1 (en) 2018-10-30 2020-05-07 Yale University Compositions and methods for rapid and modular generation of chimeric antigen receptor t cells
KR20210128440A (ko) * 2019-02-15 2021-10-26 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 범용 공여자 줄기 세포 및 관련 방법

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