CN114502731A - 基于转座子的免疫细胞的修饰 - Google Patents

基于转座子的免疫细胞的修饰 Download PDF

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Publication number
CN114502731A
CN114502731A CN202080027488.0A CN202080027488A CN114502731A CN 114502731 A CN114502731 A CN 114502731A CN 202080027488 A CN202080027488 A CN 202080027488A CN 114502731 A CN114502731 A CN 114502731A
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polynucleotide
immune cell
cells
seq
cell
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Chinese (zh)
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马克·科博尔德
玛吉·李
杰乐米·明舒尔
石丰
水一凡
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Dna20 Co ltd
General Hospital Corp
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Dna20 Co ltd
General Hospital Corp
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CN202080027488.0A 2019-02-08 2020-02-07 基于转座子的免疫细胞的修饰 Pending CN114502731A (zh)

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US201962803142P 2019-02-08 2019-02-08
US62/803,142 2019-02-08
PCT/US2020/017283 WO2020163755A1 (en) 2019-02-08 2020-02-07 Transposon-based modifications of immune cells

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CN114502731A true CN114502731A (zh) 2022-05-13

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US (1) US20220170044A1 (https=)
EP (1) EP3920941A4 (https=)
JP (1) JP2022521486A (https=)
KR (1) KR20220030205A (https=)
CN (1) CN114502731A (https=)
AU (1) AU2020218546A1 (https=)
CA (1) CA3129263A1 (https=)
IL (1) IL285422A (https=)
SG (1) SG11202108665PA (https=)
WO (1) WO2020163755A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
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CN121065098A (zh) * 2025-11-10 2025-12-05 浙江大学 一种stat3突变的细胞产品及其用途

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2020232691B2 (en) 2019-03-05 2023-06-29 Nkarta, Inc. CD19-directed chimeric antigen receptors and uses thereof in immunotherapy
US20220370495A1 (en) * 2019-08-21 2022-11-24 Board Of Regents, The University Of Texas System Immune cells for adoptive cell therapies
KR20220062305A (ko) * 2019-09-16 2022-05-16 맥마스터 유니버시티 키메라 공자극 수용체 및 이의 방법 및 용도
CN115698268A (zh) 2020-05-04 2023-02-03 萨利欧基因治疗公司 基于转座的疗法
EP4221725A4 (en) * 2020-10-02 2025-01-15 The Regents Of The University Of California DNA CONSTRUCTS FOR IMPROVED T-CELL IMMUNOTHERAPY OF CANCER
US20230392166A1 (en) * 2020-10-21 2023-12-07 Wisconsin Alumni Research Foundation Anti-apoptotic vector and method of using the same
CN115894706A (zh) * 2021-09-30 2023-04-04 北京康辰药业股份有限公司 靶向PD-L1和FasL的融合蛋白、核苷酸序列及其应用
WO2023081815A1 (en) * 2021-11-04 2023-05-11 Saliogen Therapeutics, Inc. Manufacturing of stem cells
AU2022381757A1 (en) * 2021-11-04 2024-05-09 Saliogen Therapeutics, Inc. Immune cells with chimeric antigen receptors or chimeric autoantibody receptors
JP2024541501A (ja) * 2021-11-25 2024-11-08 ドイチェス クレブスフォルシュンクスツェントルム スチフトゥング デス エッフェントリヒェン レヒツ Ｃｄ９５ポリペプチド
WO2023150801A2 (en) * 2022-02-07 2023-08-10 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Recombinant proteins that stimulate an immune response in the presence of naturally inhibitory ligand binding
EP4479067A2 (en) * 2022-02-14 2024-12-25 The Regents Of The University Of California Dna constructs for improved t cell immunotherapy
JP2025509769A (ja) * 2022-03-15 2025-04-11 ニューヨーク・ゲノム・センター，インコーポレーテッド 改善した免疫療法のための融合タンパク質を含む方法及び組成物
WO2023178358A2 (en) * 2022-03-18 2023-09-21 Dna Twopointo, Inc. Modification of t cells
WO2024233669A2 (en) * 2023-05-11 2024-11-14 Saliogen Therapeutics, Inc. Compositions and methods for treatment of cystic fibrosis
WO2025235576A1 (en) * 2024-05-08 2025-11-13 Saliogen Therapeutics, Inc. Myotis lucifugus transposase engineering

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6143291A (en) * 1995-05-04 2000-11-07 Us Navy Methods for modulating T cell survival by modulating bcl-XL protein level
CN104853766A (zh) * 2012-10-02 2015-08-19 纪念斯隆-凯特琳癌症中心 用于免疫疗法的组合物和方法
WO2017147538A1 (en) * 2016-02-26 2017-08-31 Poseida Therapeutics, Inc. Transposon system and methods of use
CN107207615A (zh) * 2014-11-05 2017-09-26 得克萨斯州大学系统董事会 基因修饰的免疫效应细胞和用于扩增免疫效应细胞的工程化细胞
EP3360961A1 (en) * 2015-10-08 2018-08-15 National University Corporation Nagoya University Method for preparing genetically-modified t cells which express chimeric antigen receptor
WO2018148440A1 (en) * 2017-02-08 2018-08-16 Dana-Farber Cancer Institute, Inc. Regulating chimeric antigen receptors

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU5673496A (en) * 1995-05-04 1996-11-21 Arch Development Corporation Methods for modulating t cell survival by modulating bcl-xl protein level
FR2736931B1 (fr) * 1995-07-17 1997-09-19 Univ Paris Curie Sequences regulatrices issues du gene cd4 et a expression specifique dans les lymphocytes t matures
AU2003231048A1 (en) * 2002-04-22 2003-11-03 Regents Of The University Of Minnesota Transposon system and methods of use
WO2010085699A2 (en) * 2009-01-23 2010-07-29 The Johns Hopkins University Mammalian piggybac transposon and methods of use
US8592211B2 (en) * 2009-03-20 2013-11-26 The Rockefeller University Enhanced PiggyBac transposon and methods for transposon mutagenesis
US10927384B2 (en) * 2014-04-09 2021-02-23 Dna Twopointo Inc. DNA vectors, transposons and transposases for eukaryotic genome modification
CA3001312A1 (en) * 2015-10-08 2017-04-13 Dna2.0, Inc. Dna vectors, transposons and transposases for eukaryotic genome modification
WO2017133175A1 (en) * 2016-02-04 2017-08-10 Nanjing Legend Biotech Co., Ltd. Engineered mammalian cells for cancer therapy
EP4043485A1 (en) * 2017-01-26 2022-08-17 Novartis AG Cd28 compositions and methods for chimeric antigen receptor therapy
AU2018236461B2 (en) * 2017-03-17 2025-03-27 Fred Hutchinson Cancer Center Immunomodulatory fusion proteins and uses thereof

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6143291A (en) * 1995-05-04 2000-11-07 Us Navy Methods for modulating T cell survival by modulating bcl-XL protein level
CN104853766A (zh) * 2012-10-02 2015-08-19 纪念斯隆-凯特琳癌症中心 用于免疫疗法的组合物和方法
CN107207615A (zh) * 2014-11-05 2017-09-26 得克萨斯州大学系统董事会 基因修饰的免疫效应细胞和用于扩增免疫效应细胞的工程化细胞
EP3360961A1 (en) * 2015-10-08 2018-08-15 National University Corporation Nagoya University Method for preparing genetically-modified t cells which express chimeric antigen receptor
WO2017147538A1 (en) * 2016-02-26 2017-08-31 Poseida Therapeutics, Inc. Transposon system and methods of use
WO2018148440A1 (en) * 2017-02-08 2018-08-16 Dana-Farber Cancer Institute, Inc. Regulating chimeric antigen receptors

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
GRZEGORZ B. GMYREK等: "Functional analysis of acquired CD28 mutations identified in cutaneous T cell lymphoma", 《CELL IMMUNOL》, vol. 319, 10 July 2017 (2017-07-10), pages 28, XP085189941, DOI: 10.1016/j.cellimm.2017.07.002 *
秦方园等: "嵌合抗原受体修饰T细胞免疫治疗肿瘤的研究进展", 《中国肿瘤生物治疗杂志》, vol. 19, no. 06, 20 December 2012 (2012-12-20), pages 648 - 651 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121065098A (zh) * 2025-11-10 2025-12-05 浙江大学 一种stat3突变的细胞产品及其用途
CN121065098B (zh) * 2025-11-10 2026-04-17 浙江大学 一种stat3突变的细胞产品及其用途

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