CN110023490B - 用于改善的免疫细胞疗法的靶向基因插入 - Google Patents

用于改善的免疫细胞疗法的靶向基因插入 Download PDF

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CN110023490B
CN110023490B CN201780072672.5A CN201780072672A CN110023490B CN 110023490 B CN110023490 B CN 110023490B CN 201780072672 A CN201780072672 A CN 201780072672A CN 110023490 B CN110023490 B CN 110023490B
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CN110023490A (zh
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布莱恩·布瑟尔
菲利普·杜沙托
亚历山大·朱利拉特
洛朗·普瓦罗
朱利安·瓦尔顿
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Cellectis SA
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CN201780072672.5A 2016-10-19 2017-10-19 用于改善的免疫细胞疗法的靶向基因插入 Active CN110023490B (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662410187P 2016-10-19 2016-10-19
US62/410,187 2016-10-19
DKPA201670840 2016-10-27
DKPA201670840 2016-10-27
PCT/EP2017/076798 WO2018073391A1 (en) 2016-10-19 2017-10-19 Targeted gene insertion for improved immune cells therapy

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CN110023490A CN110023490A (zh) 2019-07-16
CN110023490B true CN110023490B (zh) 2024-08-09

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US (3) US11873511B2 (https=)
EP (1) EP3529351A1 (https=)
JP (1) JP7274416B2 (https=)
KR (1) KR102709329B1 (https=)
CN (1) CN110023490B (https=)
AU (1) AU2017347637B2 (https=)
BR (1) BR112019007715A2 (https=)
CA (1) CA3040048A1 (https=)
IL (1) IL265953B1 (https=)
MX (1) MX2019004215A (https=)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20180214487A1 (en) 2015-07-21 2018-08-02 The Children's Medical Center Corporation Pd-l1 expressing hematopoietic stem cells and uses
US11879137B2 (en) 2017-09-22 2024-01-23 The Children's Medical Center Corporation Treatment of type 1 diabetes and autoimmune diseases or disorders
CN110841058B (zh) * 2019-11-21 2022-09-02 北京启辰生生物科技有限公司 用于协同改善免疫应答的组合物及应用
CN110790842B (zh) * 2019-11-25 2021-03-30 贵州康钦承平生物科技有限公司 一种FasL-CAR融合蛋白和表达融合蛋白的T细胞及其制备方法和应用
JP2023531531A (ja) * 2020-06-26 2023-07-24 ジュノ セラピューティクス ゲーエムベーハー 組換え受容体を条件付きで発現する操作されたt細胞、関連ポリヌクレオチド、および方法
US11459372B2 (en) 2020-11-30 2022-10-04 Crispr Therapeutics Ag Gene-edited natural killer cells
KR20230118887A (ko) 2020-12-03 2023-08-14 센츄리 쎄라퓨틱스 인코포레이티드 유전자 조작 세포 및 이의 용도
US11661459B2 (en) 2020-12-03 2023-05-30 Century Therapeutics, Inc. Artificial cell death polypeptide for chimeric antigen receptor and uses thereof
AR124414A1 (es) 2020-12-18 2023-03-22 Century Therapeutics Inc Sistema de receptor de antígeno quimérico con especificidad de receptor adaptable
WO2022144632A1 (en) 2020-12-30 2022-07-07 Crispr Therapeutics Ag Compositions and methods for differentiating stem cells into nk cells
KR102491095B1 (ko) * 2021-01-22 2023-01-20 한국생명공학연구원 Cas 단백질 및 HIF의 TAD 도메인을 포함하는 단백질 융합체 및 이의 용도
KR102500873B1 (ko) * 2021-02-03 2023-02-17 한국과학기술연구원 자연살해세포 특이적 CRISPR/Cas 시스템을 위한 융합 단백질 및 이의 용도
WO2022269393A1 (en) * 2021-06-23 2022-12-29 Crispr Therapeutics Ag Engineered cells with improved protection from natural killer cell killing
CN113604437A (zh) * 2021-08-13 2021-11-05 青岛华赛伯曼医学细胞生物有限公司 过表达ccr2的免疫细胞及其应用
CN118234851A (zh) * 2021-11-19 2024-06-21 南开大学 重组武装溶瘤病毒组合物及其在til过继治疗中的用途
WO2024022509A1 (en) * 2022-07-29 2024-02-01 Nanjing Legend Biotech Co., Ltd. Methods for promoting persistence of cell therapy
CN115820697B (zh) * 2022-09-23 2024-07-30 中国海洋大学 一种免疫细胞及其制备方法和应用
CN115286697B (zh) * 2022-09-29 2022-12-13 烟台蓝纳成生物技术有限公司 一种双重靶向化合物及其制备方法和应用
CN115851909B (zh) * 2022-11-21 2023-09-08 郑州大学第一附属医院 检测fosb基因的试剂在制备诊断腹主动脉瘤产品的应用

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112014029417B1 (pt) * 2012-05-25 2023-03-07 Cellectis Método ex vivo para a preparação de células t para imunoterapia
AU2014266833B2 (en) * 2013-05-13 2020-07-02 Cellectis Methods for engineering highly active T cell for immunotherapy
WO2014191128A1 (en) * 2013-05-29 2014-12-04 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
WO2015075195A1 (en) * 2013-11-22 2015-05-28 Cellectis Method of engineering chemotherapy drug resistant t-cells for immunotherapy
JP6681837B2 (ja) 2014-03-11 2020-04-15 セレクティスCellectis 同種移植に適合するt細胞を作製するための方法
WO2016124765A1 (en) 2015-02-06 2016-08-11 Cellectis Primary hematopoietic cells genetically engineered by slow release of nucleic acids using nanoparticles
AU2016243583B2 (en) 2015-03-27 2022-03-10 President And Fellows Of Harvard College Modified T cells and methods of making and using the same
GB2592821B (en) * 2015-07-31 2022-01-12 Univ Minnesota Modified cells and methods of therapy
EP4628587A3 (en) * 2016-04-15 2026-02-25 Memorial Sloan Kettering Cancer Center Transgenic t cell and chimeric antigen receptor t cell compositions and related methods

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RU2019113911A (ru) 2020-11-20
JP7274416B2 (ja) 2023-05-16
AU2017347637B2 (en) 2024-02-15
KR20190065397A (ko) 2019-06-11
JP2019531743A (ja) 2019-11-07
US20240141293A1 (en) 2024-05-02
MX2019004215A (es) 2019-08-21
CN110023490A (zh) 2019-07-16
BR112019007715A2 (pt) 2019-07-09
US11873511B2 (en) 2024-01-16
EP3529351A1 (en) 2019-08-28
RU2019113911A3 (https=) 2021-06-28
CA3040048A1 (en) 2018-04-26
IL265953B1 (en) 2026-01-01
AU2017347637A1 (en) 2019-05-02
KR102709329B1 (ko) 2024-09-23
US20200224163A1 (en) 2020-07-16
US12391933B2 (en) 2025-08-19
IL265953A (en) 2019-05-30
US20260103694A1 (en) 2026-04-16

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