JP7432362B2 - modRNAの細胞特異的発現 - Google Patents

modRNAの細胞特異的発現 Download PDF

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JP7432362B2
JP7432362B2 JP2019514770A JP2019514770A JP7432362B2 JP 7432362 B2 JP7432362 B2 JP 7432362B2 JP 2019514770 A JP2019514770 A JP 2019514770A JP 2019514770 A JP2019514770 A JP 2019514770A JP 7432362 B2 JP7432362 B2 JP 7432362B2
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ザンギ,リオール
マガダム,アジット
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Icahn School of Medicine at Mount Sinai
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JP2019514770A 2016-09-16 2017-09-18 modRNAの細胞特異的発現 Active JP7432362B2 (ja)

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JP2023093720A JP2023123520A (ja) 2016-09-16 2023-06-07 modRNAの細胞特異的発現

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US201662395701P 2016-09-16 2016-09-16
US62/395,701 2016-09-16
PCT/US2017/052035 WO2018053414A1 (en) 2016-09-16 2017-09-18 Cell-specific expression of modrna

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JP2019528736A JP2019528736A (ja) 2019-10-17
JP2019528736A5 JP2019528736A5 (https=) 2020-11-12
JP7432362B2 true JP7432362B2 (ja) 2024-02-16

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EP (2) EP3512567B1 (https=)
JP (2) JP7432362B2 (https=)
CN (1) CN110177578B (https=)
AU (2) AU2017326535B2 (https=)
BR (1) BR112019005144A2 (https=)
CA (1) CA3036710A1 (https=)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023123520A (ja) * 2016-09-16 2023-09-05 アイカーン スクール オブ メディスン アット マウント サイナイ modRNAの細胞特異的発現

Families Citing this family (15)

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Publication number Priority date Publication date Assignee Title
JP7440868B2 (ja) * 2018-03-30 2024-02-29 国立大学法人京都大学 細胞の製造方法
BR112022002365A2 (pt) 2019-08-09 2022-04-26 Nutcracker Therapeutics Inc Métodos e aparelhos para fabricar e para remover material de uma composição terapêutica
WO2021092440A1 (en) 2019-11-07 2021-05-14 Icahn School Of Medicine At Mount Sinai Synthetic modified rna and uses thereof
EP4058584A4 (en) * 2019-11-11 2023-12-20 Icahn School of Medicine at Mount Sinai GENE DELIVERY SYSTEMS FOR THE TREATMENT OF HEART FAILURE
EP4351623A1 (en) 2021-06-09 2024-04-17 Icahn School of Medicine at Mount Sinai Gene combination as a broad spectrum antiviral
EP4355882A2 (en) * 2021-06-15 2024-04-24 Modernatx, Inc. Engineered polynucleotides for cell-type or microenvironment-specific expression
CN114085834A (zh) * 2021-11-16 2022-02-25 珠海中科先进技术研究院有限公司 一种癌细胞导向电路组及应用
EP4514965A1 (en) * 2022-04-27 2025-03-05 The Penn State Research Foundation Modrna-based cas endonuclease and base editor and uses thereof
US20230364267A1 (en) * 2022-05-13 2023-11-16 The Uab Research Foundation Pre-clinical modified rna approaches used in large animals for muscle and vascular regeneration
EP4282963A1 (en) * 2022-05-23 2023-11-29 Eberhard Karls Universität Tübingen, Medizinische Fakultät Nucleic acid modified biological cell with expansion-dependent gene expression
WO2024015729A1 (en) * 2022-07-11 2024-01-18 Icahn School Of Medicine At Mount Sinai Regulatory system for expression of a gene of interest in a target cell and method of use thereof
US20260035707A1 (en) * 2022-07-26 2026-02-05 Modernatx, Inc. Engineered polynucleotides for temporal control of expression
CN116785501A (zh) * 2022-08-04 2023-09-22 上海交通大学医学院附属上海儿童医学中心 促进心脏组织修复的modRNA转染改造的细胞及其应用
WO2024151975A1 (en) 2023-01-13 2024-07-18 Igor Lednev Detecting degradation of a polynucleotide by raman spectroscopy
CN119120571B (zh) * 2024-09-11 2026-03-17 武汉科技大学 一种治疗hiv感染的靶向外泌体嵌合抗原受体分子的构建及其应用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015141827A1 (ja) 2014-03-20 2015-09-24 国立大学法人京都大学 心筋細胞の選別方法
WO2016040395A1 (en) 2014-09-08 2016-03-17 Massachusetts Institute Of Technology Rna-based logic circuits with rna binding proteins, aptamers and small molecules

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH08506008A (ja) 1992-11-18 1996-07-02 アーチ ディベロプメント コーポレイション 心筋及び血管平滑筋へのアデノウィルス介在遺伝子移入
US20090005336A1 (en) * 2007-05-08 2009-01-01 Zhiguo Wang Use of the microRNA miR-1 for the treatment, prevention, and diagnosis of cardiac conditions
KR20240136456A (ko) * 2009-12-07 2024-09-13 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 세포 리프로그래밍을 위한 정제된 변형 rna를 포함하는 rna 제제
US9458509B2 (en) * 2010-07-22 2016-10-04 President And Fellows Of Harvard College Multiple input biologic classifier circuits for cells
EP2694660B1 (en) 2011-04-03 2018-08-08 The General Hospital Corporation Efficient protein expression in vivo using modified rna (mod-rna)
EP2584040A1 (en) * 2011-10-17 2013-04-24 Pharmahungary 2000 Kft. Compounds for treatment of ischemic injury
CA2915627A1 (en) * 2012-06-15 2013-12-19 The General Hospital Corporation Inhibitors of micrornas that regulate production of atrial natriuretic peptide (anp) as therapeutics and uses thereof
WO2014093574A1 (en) * 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Modified polynucleotides for altering cell phenotype
EP3512567B1 (en) * 2016-09-16 2023-11-01 Icahn School of Medicine at Mount Sinai Cell-specific expression of modrna

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015141827A1 (ja) 2014-03-20 2015-09-24 国立大学法人京都大学 心筋細胞の選別方法
WO2016040395A1 (en) 2014-09-08 2016-03-17 Massachusetts Institute Of Technology Rna-based logic circuits with rna binding proteins, aptamers and small molecules

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
Biochem. Biophys. Res. Commun.,2016年01月,Vol. 469,p. 29-36
Cell Stem Cell,2015年,Vol. 16,p. 699-711
J. Cell. Mol. Med.,2015年,Vol. 19,p, 1174-1182
Nat. Biotechnol.,2015年,Vol. 33,p. 839-841

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023123520A (ja) * 2016-09-16 2023-09-05 アイカーン スクール オブ メディスン アット マウント サイナイ modRNAの細胞特異的発現

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EP3512567B1 (en) 2023-11-01
JP2019528736A (ja) 2019-10-17
CA3036710A1 (en) 2018-03-22
CN110177578B (zh) 2024-02-13
US12215336B2 (en) 2025-02-04
US20190203226A1 (en) 2019-07-04
EP3512567A4 (en) 2020-03-18
US20220220501A1 (en) 2022-07-14
AU2017326535B2 (en) 2023-08-24
EP4316587A3 (en) 2024-04-24
CN110177578A (zh) 2019-08-27
BR112019005144A2 (pt) 2019-06-04
AU2017326535A1 (en) 2019-04-18
US11299749B2 (en) 2022-04-12
US20250154523A1 (en) 2025-05-15
WO2018053414A1 (en) 2018-03-22
ES2967788T3 (es) 2024-05-03
EP4316587A2 (en) 2024-02-07
EP3512567A1 (en) 2019-07-24
EP3512567C0 (en) 2023-11-01
AU2023270313A1 (en) 2024-01-04

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