CN110785488A - 基因疗法 - Google Patents

基因疗法 Download PDF

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Publication number
CN110785488A
CN110785488A CN201880040639.9A CN201880040639A CN110785488A CN 110785488 A CN110785488 A CN 110785488A CN 201880040639 A CN201880040639 A CN 201880040639A CN 110785488 A CN110785488 A CN 110785488A
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Prior art keywords
ifn
cells
mir
cancer
myeloid
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CN201880040639.9A
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English (en)
Chinese (zh)
Inventor
L.纳尔蒂尼
G.埃斯科巴尔
B.R.金特纳
A.穆西
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Ospedale San Raffaele SRL
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Ospedale San Raffaele SRL
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Priority claimed from GBGB1706410.6A external-priority patent/GB201706410D0/en
Priority claimed from GBGB1801511.5A external-priority patent/GB201801511D0/en
Application filed by Ospedale San Raffaele SRL filed Critical Ospedale San Raffaele SRL
Publication of CN110785488A publication Critical patent/CN110785488A/zh
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CN201880040639.9A 2017-04-21 2018-04-20 基因疗法 Pending CN110785488A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GB1706410.6 2017-04-21
GBGB1706410.6A GB201706410D0 (en) 2017-04-21 2017-04-21 Type 1 interferon gene therapy
GBGB1801511.5A GB201801511D0 (en) 2018-01-30 2018-01-30 Gene therapy
GB1801511.5 2018-01-30
PCT/EP2018/060238 WO2018193119A1 (en) 2017-04-21 2018-04-20 Gene therapy

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Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112280829A (zh) * 2020-04-07 2021-01-29 南方科技大学 一种试剂盒、样本标记方法、单细胞测序方法
CN114426953A (zh) * 2020-10-29 2022-05-03 未来智人再生医学研究院(广州)有限公司 一种表达il-12的多能干细胞衍生物及应用
WO2022095642A1 (zh) * 2020-11-03 2022-05-12 广州辑因医疗科技有限公司 细胞制剂、蛋白在表征造血干细胞中的用途及判定造血干细胞的方法
WO2022152266A1 (zh) * 2021-01-18 2022-07-21 上海邦耀生物科技有限公司 用于基因编辑的组合物及其应用
CN115867569A (zh) * 2020-05-14 2023-03-28 圣拉斐尔医院有限责任公司 经修饰的表皮生长因子受体及其在追踪细胞中的用途
CN115968300A (zh) * 2020-05-11 2023-04-14 艾宾妥斯生物公司 用于体内转导的载体和方法

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020163868A1 (en) * 2019-02-09 2020-08-13 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Genetically modified hematopoietic stem and progenitor cells (hspcs) and mesenchymal cells as a platform to reduce or prevent metastasis, treat autoimmune and inflammatory disorders, and rebalance the immune milieu and dysregulated niches
AU2020225917A1 (en) * 2019-02-22 2021-08-26 Oncolytics Biotech Inc. T cell repertoire dynamics and oncolytic viral therapy
WO2020191358A1 (en) * 2019-03-20 2020-09-24 Bluebird Bio, Inc. Adoptive cell therapy
EP4161536A4 (en) * 2020-06-04 2024-08-14 Carisma Therapeutics Inc. Novel constructs for chimeric antigen receptors
WO2024068617A1 (en) * 2022-09-26 2024-04-04 Institut Curie Myeloid cells expressing il-2 and uses thereof for quick anticancer therapy
CN120603934A (zh) * 2023-02-08 2025-09-05 国立大学法人九州大学 能够在肿瘤组织内分化成m2巨噬细胞且分化成m2巨噬细胞时表达炎性细胞因子的细胞和含有该细胞的组合物
WO2024194223A2 (en) * 2023-03-17 2024-09-26 Ospedale San Raffaele S.R.L. Gene therapy
WO2025217427A1 (en) * 2024-04-11 2025-10-16 Genenta Science S.P.A. Combination immunotherapy of solid cancers

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999041397A1 (en) * 1998-02-17 1999-08-19 Oxford Biomedica (Uk) Limited Anti-viral vectors
CN102596255A (zh) * 2009-04-30 2012-07-18 圣拉斐尔德尔蒙特塔博基金中心 基因载体
US20140200261A1 (en) * 2013-01-17 2014-07-17 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB0009760D0 (en) 2000-04-19 2000-06-07 Oxford Biomedica Ltd Method
PT2856876T (pt) * 2007-03-30 2018-03-28 Memorial Sloan Kettering Cancer Center Expressão constitutiva de ligantes co-estimulatórios em linfócitos t adotivamente transferidos
EP3347375B1 (en) * 2015-09-10 2020-12-23 The United States of America, as represented by The Secretary, Department of Health and Human Services Anti-cd276 chimeric antigen receptors
JP2018536436A (ja) * 2015-12-04 2018-12-13 ノバルティス アーゲー 免疫腫瘍学のための組成物および方法

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999041397A1 (en) * 1998-02-17 1999-08-19 Oxford Biomedica (Uk) Limited Anti-viral vectors
CN102596255A (zh) * 2009-04-30 2012-07-18 圣拉斐尔德尔蒙特塔博基金中心 基因载体
JP2012525141A (ja) * 2009-04-30 2012-10-22 フォンダツィオーネ セントロ サン ラファエレ デル モンテ タボール 遺伝子ベクター
US20140200261A1 (en) * 2013-01-17 2014-07-17 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ESCOBAR， G: ""Genetic Engineering of Hematopoiesis for Targeted IFN-Delivery Inhibits Breast Cancer Progression"", 《SCIENCE TRANSLATIONAL MEDICINE》 *
GENTNER， B: ""Identification of Hematopoietic Stem Cell-Specific miRNAs Enables Gene Therapy of Globoid Cell Leukodystrophy"", 《SCIENCE TRANSLATIONAL MEDICINE》 *

Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112280829A (zh) * 2020-04-07 2021-01-29 南方科技大学 一种试剂盒、样本标记方法、单细胞测序方法
CN112280829B (zh) * 2020-04-07 2023-12-26 南方科技大学 一种试剂盒、样本标记方法、单细胞测序方法
CN115968300A (zh) * 2020-05-11 2023-04-14 艾宾妥斯生物公司 用于体内转导的载体和方法
CN115867569A (zh) * 2020-05-14 2023-03-28 圣拉斐尔医院有限责任公司 经修饰的表皮生长因子受体及其在追踪细胞中的用途
CN114426953A (zh) * 2020-10-29 2022-05-03 未来智人再生医学研究院(广州)有限公司 一种表达il-12的多能干细胞衍生物及应用
WO2022095642A1 (zh) * 2020-11-03 2022-05-12 广州辑因医疗科技有限公司 细胞制剂、蛋白在表征造血干细胞中的用途及判定造血干细胞的方法
WO2022152266A1 (zh) * 2021-01-18 2022-07-21 上海邦耀生物科技有限公司 用于基因编辑的组合物及其应用

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