JP2019528284A5 - - Google Patents

Download PDF

Info

Publication number
JP2019528284A5
JP2019528284A5 JP2019509510A JP2019509510A JP2019528284A5 JP 2019528284 A5 JP2019528284 A5 JP 2019528284A5 JP 2019509510 A JP2019509510 A JP 2019509510A JP 2019509510 A JP2019509510 A JP 2019509510A JP 2019528284 A5 JP2019528284 A5 JP 2019528284A5
Authority
JP
Japan
Prior art keywords
composition
uridine
utr
sequence
synthetic
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2019509510A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019528284A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2017/047440 external-priority patent/WO2018035377A1/en
Publication of JP2019528284A publication Critical patent/JP2019528284A/ja
Publication of JP2019528284A5 publication Critical patent/JP2019528284A5/ja
Priority to JP2022168829A priority Critical patent/JP2023011696A/ja
Priority to JP2022168830A priority patent/JP2023011697A/ja
Pending legal-status Critical Current

Links

JP2019509510A 2016-08-17 2017-08-17 核酸産物およびその投与方法 Pending JP2019528284A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022168829A JP2023011696A (ja) 2016-08-17 2022-10-21 核酸産物およびその投与方法
JP2022168830A JP2023011697A (ja) 2016-08-17 2022-10-21 核酸産物およびその投与方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662376209P 2016-08-17 2016-08-17
US62/376,209 2016-08-17
US201762509350P 2017-05-22 2017-05-22
US62/509,350 2017-05-22
PCT/US2017/047440 WO2018035377A1 (en) 2016-08-17 2017-08-17 Nucleic acid products and methods of administration thereof

Related Child Applications (2)

Application Number Title Priority Date Filing Date
JP2022168830A Division JP2023011697A (ja) 2016-08-17 2022-10-21 核酸産物およびその投与方法
JP2022168829A Division JP2023011696A (ja) 2016-08-17 2022-10-21 核酸産物およびその投与方法

Publications (2)

Publication Number Publication Date
JP2019528284A JP2019528284A (ja) 2019-10-10
JP2019528284A5 true JP2019528284A5 (https=) 2020-09-24

Family

ID=61197041

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2019509510A Pending JP2019528284A (ja) 2016-08-17 2017-08-17 核酸産物およびその投与方法
JP2022168830A Pending JP2023011697A (ja) 2016-08-17 2022-10-21 核酸産物およびその投与方法
JP2022168829A Pending JP2023011696A (ja) 2016-08-17 2022-10-21 核酸産物およびその投与方法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2022168830A Pending JP2023011697A (ja) 2016-08-17 2022-10-21 核酸産物およびその投与方法
JP2022168829A Pending JP2023011696A (ja) 2016-08-17 2022-10-21 核酸産物およびその投与方法

Country Status (8)

Country Link
US (12) US10576167B2 (https=)
EP (1) EP3500585A4 (https=)
JP (3) JP2019528284A (https=)
CN (2) CN109803977B (https=)
AU (2) AU2017312113B2 (https=)
CA (1) CA3033788A1 (https=)
IL (2) IL264439B2 (https=)
WO (1) WO2018035377A1 (https=)

Families Citing this family (59)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2214701T3 (en) * 2007-11-01 2016-12-12 Univ Arkansas CONFIGURATIONS AND METHODS FOR IMPROVING THE IMMUNE RESPONSES TO Eimeria
WO2014071219A1 (en) * 2012-11-01 2014-05-08 Factor Bioscience Inc. Methods and products for expressing proteins in cells
US11111505B2 (en) 2016-03-19 2021-09-07 Exuma Biotech, Corp. Methods and compositions for transducing lymphocytes and regulating the activity thereof
US11325948B2 (en) 2016-03-19 2022-05-10 Exuma Biotech Corp. Methods and compositions for genetically modifying lymphocytes to express polypeptides comprising the intracellular domain of MPL
US12590321B2 (en) 2016-03-19 2026-03-31 Exuma Biotech Corp. Methods and compositions for genetically modifying and expanding lymphocytes and regulating the activity thereof
US10576167B2 (en) * 2016-08-17 2020-03-03 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
BR112019018915A2 (pt) 2017-03-15 2020-04-14 Pandion Therapeutics Inc imunotolerância direcionada
CN106987604B (zh) * 2017-03-29 2021-05-28 北京希诺谷生物科技有限公司 一种制备动脉粥样硬化疾病模型犬的方法
JP2020521452A (ja) 2017-05-24 2020-07-27 パンディオン・セラピューティクス・インコーポレイテッド 標的化免疫寛容
WO2018222926A1 (en) 2017-05-31 2018-12-06 Ultragenyx Pharmaceutical Inc. Therapeutics for glycogen storage disease type iii
EP3714048B1 (en) 2017-11-22 2025-04-09 ModernaTX, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
US10174091B1 (en) 2017-12-06 2019-01-08 Pandion Therapeutics, Inc. IL-2 muteins
USRE50550E1 (en) 2017-12-06 2025-08-26 Pandion Operations, Inc. IL-2 muteins and uses thereof
US10946068B2 (en) 2017-12-06 2021-03-16 Pandion Operations, Inc. IL-2 muteins and uses thereof
CA3095125A1 (en) * 2018-03-27 2019-10-03 Factor Bioscience Inc. Nucleic acid-based therapeutics
JP7558929B2 (ja) * 2018-05-11 2024-10-01 ビーム セラピューティクス インク. プログラム可能塩基エディターシステムを用いて病原性変異を抑制する方法
CN109468372B (zh) * 2018-11-21 2020-08-04 中国人民解放军陆军军医大学第一附属医院 多种遗传代谢性肝病靶向文库的引物组合、方法及试剂盒
WO2020117715A1 (en) 2018-12-03 2020-06-11 Board Of Regents, The University Of Texas System Oligo-benzamide analogs and their use in cancer treatment
JP7445657B2 (ja) 2018-12-06 2024-03-07 アークトゥラス・セラピューティクス・インコーポレイテッド オルニチントランスカルバミラーゼ欠損症を処置するための組成物および方法
CN109750038B (zh) * 2018-12-29 2021-08-31 烟台毓璜顶医院 一种长非编码rna及在制备诊断子痫前期及靶点药物治疗中的应用
CN109628454B (zh) * 2019-01-30 2022-05-24 上海海洋大学 斑马鱼糖原贮积症gys1和gys2基因突变体的构建方法
CN109777832B (zh) * 2019-02-01 2020-08-04 国家卫生健康委科学技术研究所 碱基编辑模拟、修复与甘露糖苷贮积症相关的man2b1c2248t突变的试剂和方法
WO2020236875A1 (en) 2019-05-20 2020-11-26 Pandion Therapeutics, Inc. Madcam targeted immunotolerance
WO2020252455A1 (en) 2019-06-13 2020-12-17 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
CN110250109B (zh) * 2019-07-01 2021-09-24 上海交通大学医学院附属新华医院 乙醛酸代谢异常相关疾病模型的构建方法、组合物及试剂盒和应用
JP7699552B2 (ja) 2019-07-03 2025-06-27 ファクター バイオサイエンス インコーポレイテッド カチオン性脂質及びその使用
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
BR112022002365A2 (pt) 2019-08-09 2022-04-26 Nutcracker Therapeutics Inc Métodos e aparelhos para fabricar e para remover material de uma composição terapêutica
JP2022546101A (ja) * 2019-09-01 2022-11-02 エクスマ バイオテック コーポレイション リンパ球の改変および送達のための方法および組成物
CN111100196B (zh) * 2019-11-08 2021-07-13 上海交通大学 一种生物活性多肽qilsvpgwtysr及其制备方法和应用
EP4067495A4 (en) * 2019-11-26 2023-06-14 Korea Research Institute of Bioscience and Biotechnology MRNA CONSTRUCT FOR PROTEIN EXPRESSION AND ITS USE
US11981715B2 (en) 2020-02-21 2024-05-14 Pandion Operations, Inc. Tissue targeted immunotolerance with a CD39 effector
US12606868B2 (en) 2020-02-24 2026-04-21 The Board Of Regents Of The University Of Texas System Molecular biomarkers and targets for Fuchs' endothelial corneal dystrophy and glaucoma
EP4110355A1 (en) * 2020-02-24 2023-01-04 The United States of America, as represented by the Secretary, Department of Health and Human Services Nk cells or t cells expressing hematopoietic growth factor receptors and use for treating cancer
WO2021178448A1 (en) * 2020-03-02 2021-09-10 Motor Life Sciences, Llc Compositions and methods for diagnosing, preventing, and treating amyotrophic lateral sclerosis in patients with hypofunctional anti-trypsin activity
JP7832114B2 (ja) 2020-03-03 2026-03-17 アークトゥラス・セラピューティクス・インコーポレイテッド オルニチントランスカルバミラーゼ欠損症の治療のための組成物及び方法
WO2021231549A2 (en) * 2020-05-12 2021-11-18 Factor Bioscience Inc. Engineered gene-editing proteins
CN111714619B (zh) * 2020-07-15 2023-03-14 上海市浦东新区人民医院 成纤维生长因子6在制备缓解非酒精性脂肪性肝炎肝损伤的药物中的应用
EP4185265A4 (en) 2020-07-21 2024-06-26 Chembeau LLC DIESTER COSMETIC FORMULATIONS AND USES THEREOF
EP4185600A4 (en) 2020-07-24 2024-12-04 The General Hospital Corporation Enhanced virus-like particles and methods of use thereof for delivery to cells
CN111944094B (zh) * 2020-08-07 2022-01-07 西南石油大学 水凝胶共聚物、油水分离涂料及其制备方法、应用、油水分离网及其制备方法
CN112143789A (zh) * 2020-09-10 2020-12-29 长沙金域医学检验实验室有限公司 一种鉴定galt基因同时含两个突变位点位置的试剂盒及方法
WO2022125548A1 (en) * 2020-12-08 2022-06-16 The Board Of Trustees Of The Leland Stanford Junior University Growth and differentiation factor 15 for treatment of proliferative vitreoretinopathy therapy
CN114717229B (zh) * 2021-01-05 2024-09-10 麦塞拿治疗(香港)有限公司 治疗性mRNA的无细胞和无载体体外RNA转录方法和核酸分子
US12364531B2 (en) * 2021-02-16 2025-07-22 RecensMedical, Inc.; Methods for treating skin disorders using precision cooling technology
BR112023020209A2 (pt) 2021-04-02 2023-12-19 Krystal Biotech Inc Genoma do vírus do herpes recombinante, vírus do herpes, composição farmacêutica, uso do vírus do herpes ou da composição farmacêutica, método para expressar, realçar, aumentar, ampliar e/ou suplementar os níveis de um polipeptídeo imunomodulatório, método para prover alívio profilático, paliativo ou terapêutico, e, método para tratar câncer
CN113151182B (zh) * 2021-04-19 2022-12-06 深圳明杰生物工程有限公司 基于msc的基因重组细胞的制备方法及其应用
CA3213739A1 (en) * 2021-05-03 2022-09-15 Nikolay ZHUKOVSKY Alpha-1-antitrypsin (aat) in the treatment and/or prevention of neurological disorders
KR20240006603A (ko) 2021-05-07 2024-01-15 헬릭스 나노테크놀로지스, 인코포레이티드 변형된 리보핵산 및 이의 용도
CA3231594A1 (en) * 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Serpina-modulating compositions and methods
EP4466005A1 (en) * 2022-01-21 2024-11-27 Institut Pasteur De Montevideo Active immunization for reducing osteoarthritic, neuropathic, and cancer pain
CN120303407A (zh) 2022-05-17 2025-07-11 恩维洛普治疗有限责任公司 用于有效体内递送的组合物和方法
WO2024121160A1 (en) * 2022-12-05 2024-06-13 Ethris Gmbh Regulator(s) of energy homeostasis-encoding rna molecule(s) with increased translation efficiency
WO2024229116A2 (en) * 2023-05-01 2024-11-07 Factor Bioscience Inc. Methods for reprogramming and gene editing cells
CN119055671A (zh) * 2023-05-23 2024-12-03 源生生物科技(青岛)有限责任公司 包含miRNA的活性成分及其应用
CN117248020B (zh) * 2023-09-28 2025-06-27 山东大学 Hmgcl作为胶质瘤诊断/预后标志物和治疗靶点的应用
WO2025155772A1 (en) * 2024-01-19 2025-07-24 Gene Company (Pty)Ltd. Alpha-1 anti-trypsin upregulating polynucleotides and method of use and treatment thereof
CN118716283B (zh) * 2024-07-29 2025-01-28 安徽省农业科学院水产研究所 一种保持遗传多样性的鱼类育种方法
CN121476595B (zh) * 2026-01-07 2026-04-10 北京大学人民医院 抗pccb抗体在制备干燥综合征诊断产品中的应用

Family Cites Families (186)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US527565A (en) * 1894-10-16 tripp
US3034507A (en) 1960-05-10 1962-05-15 American Cyanamid Co Intracutaneous injection device
US3539465A (en) 1968-10-08 1970-11-10 Ncr Co Encapsulation of hydrophilic liquid-in-oil emulsions
US3675766A (en) 1970-02-04 1972-07-11 Sol Roy Rosenthal Multiple puncture injector device
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
IE61148B1 (en) * 1988-03-10 1994-10-05 Ici Plc Method of detecting nucleotide sequences
US5674844A (en) 1991-03-11 1997-10-07 Creative Biomolecules, Inc. Treatment to prevent loss of and/or increase bone mass in metabolic bone diseases
US6506729B1 (en) 1991-03-11 2003-01-14 Curis, Inc. Methods and compositions for the treatment and prevention of Parkinson's disease
US5849686A (en) 1991-03-11 1998-12-15 Creative Biomolecules, Inc. Morphogen-induced liver regeneration
US5656593A (en) 1991-03-11 1997-08-12 Creative Biomolecules, Inc. Morphogen induced periodontal tissue regeneration
US6800603B2 (en) 1991-03-11 2004-10-05 Curis, Inc. Morphogen-induced neural cell adhesion
US6949505B1 (en) 1991-03-11 2005-09-27 Curis, Inc. Morphogen-induced dendritic growth
US5993434A (en) 1993-04-01 1999-11-30 Genetronics, Inc. Method of treatment using electroporation mediated delivery of drugs and genes
US5843780A (en) 1995-01-20 1998-12-01 Wisconsin Alumni Research Foundation Primate embryonic stem cells
US5711964A (en) 1995-06-07 1998-01-27 United States Of America Method for the intracellular delivery of biomolecules using liposomes containing cationic lipids and vitamin D
KR20000064752A (ko) 1996-03-22 2000-11-06 더 제네랄 호스피탈 코포레이션 중추신경계허혈또는외상의발현후폴리펩티드성장인자를투여하는방법
US6498142B1 (en) 1996-05-06 2002-12-24 Curis, Inc. Morphogen treatment for chronic renal failure
US6602693B1 (en) 1996-07-03 2003-08-05 Clear Solutions Biotech, Inc. Gene encoding hyaluronan synthase
US6413544B1 (en) 1996-08-19 2002-07-02 The United States Of America As Represented By The Department Of Health And Human Services Liposome complexes for increased systemic delivery
US6770291B2 (en) 1996-08-30 2004-08-03 The United States Of America As Represented By The Department Of Health And Human Services Liposome complexes for increased systemic delivery
TW520297B (en) 1996-10-11 2003-02-11 Sequus Pharm Inc Fusogenic liposome composition and method
US6653104B2 (en) 1996-10-17 2003-11-25 Immunomedics, Inc. Immunotoxins, comprising an internalizing antibody, directed against malignant and normal cells
CA2277278A1 (en) 1997-01-10 1998-07-16 Life Technologies, Inc. Embryonic stem cell serum replacement
US6316260B1 (en) 1997-08-22 2001-11-13 Bernina Biosystems Gmbh Tetraether lipid derivatives and liposomes and lipid agglomerates containing tetraether lipid derivatives, and use thereof
US20030083272A1 (en) 1997-09-19 2003-05-01 Lahive & Cockfield, Llp Sense mrna therapy
DK1129064T3 (da) 1998-11-12 2008-04-28 Invitrogen Corp Transfektionsreagenser
GB9902000D0 (en) 1999-01-30 1999-03-17 Delta Biotechnology Ltd Process
US7390780B2 (en) 1999-04-23 2008-06-24 Alza Corporation Gene delivery mediated by liposome-DNA complex with cleavable peg surface modification
EP1187653B1 (en) 1999-06-04 2010-03-31 Georgia Tech Research Corporation Devices for enhanced microneedle penetration of biological barriers
US20110171185A1 (en) 1999-06-30 2011-07-14 Klimanskaya Irina V Genetically intact induced pluripotent cells or transdifferentiated cells and methods for the production thereof
US20020054895A1 (en) 1999-07-23 2002-05-09 Alwyn Company, Inc. Allantoin-containing skin cream
US6595947B1 (en) 2000-05-22 2003-07-22 Becton, Dickinson And Company Topical delivery of vaccines
AU9475001A (en) 2000-09-26 2002-04-08 Hybridon Inc Modulation of immunostimulatory activity of immunostimulatory oligonucleotide analogs by positional chemical changes
US7189759B2 (en) 2001-05-23 2007-03-13 Medicis Pharmaceutical Corporation Compositions for the treatment of pigmentation disorders and methods for their manufacture
AU2002313817A1 (en) 2001-08-27 2003-03-10 Advanced Cell Technology, Inc. Trans-differentiation and re-differentiation of somatic cells and production of cells for cell therapies
CA2461185A1 (en) 2001-09-21 2003-04-03 Japan Science And Technology Corporation Method of screening reprogramming factor, reprogramming factor screened by the method, method of using the reprogramming factor, method of differentiating undifferentiated fused cells and method of constructing cell, tissues and organs
CA2463914A1 (en) 2001-10-18 2003-04-24 Ixion Biotechnology, Inc. Conversion of liver stem and progenitor cells to pancreatic functional cells
US7276489B2 (en) 2002-10-24 2007-10-02 Idera Pharmaceuticals, Inc. Modulation of immunostimulatory properties of oligonucleotide-based compounds by optimal presentation of 5′ ends
GB0202149D0 (en) 2002-01-30 2002-03-20 Univ Edinburgh Pluripotency determining factors and uses thereof
US20030186249A1 (en) 2002-04-01 2003-10-02 Zairen Sun Human TARPP genes and polypeptides
JP3785508B2 (ja) 2002-04-15 2006-06-14 学校法人慶應義塾 遺伝子治療における免疫応答を解析できる実験モデルマウス
DE10234192B4 (de) 2002-07-26 2009-11-26 Epoplus Gmbh Co.Kg Verwendung von Erythropoetin
US7737182B2 (en) 2002-08-09 2010-06-15 Taisho Pharmaceutical Co., Ltd. Pharmaceuticals for xerosis
BRPI0410886A (pt) 2003-06-03 2006-07-04 Isis Pharmaceuticals Inc composto de filamento duplo, composição farmacêutica, sal farmaceuticamente aceitável, métodos de modificação do ácido nucleico que codifica a survivina humana, de inibição da expressão da suvivina em células ou tecidos, e de tratamento de uma condição associada com a expressão ou superexpressão da suvivina, e, oligonucleotìdeo de rnai de filamento único
US7682828B2 (en) 2003-11-26 2010-03-23 Whitehead Institute For Biomedical Research Methods for reprogramming somatic cells
AU2005282414C1 (en) 2004-09-08 2011-04-07 Wisconsin Alumni Research Foundation Culturing human embryonic stem cells
CN101052712A (zh) 2004-09-08 2007-10-10 威斯康星校友研究基金会 培养人胚胎干细胞
WO2006047728A2 (en) 2004-10-27 2006-05-04 New York Society For The Ruptured And Crippled Maintaining The Hospital For Special Surgery Bmp gene and fusion protein
WO2006081249A2 (en) 2005-01-25 2006-08-03 Cell Therapeutics, Inc. Conjugates of biologically active proteins having a modified in vivo half-life
ES2397851T3 (es) 2005-07-13 2013-03-11 Novo Nordisk Health Care Ag Células de inactivación proteínica de la célula huésped para la producción de proteínas terapéuticas
AU2006272634B2 (en) 2005-07-26 2013-01-24 Sangamo Therapeutics, Inc. Targeted integration and expression of exogenous nucleic acid sequences
US8323666B2 (en) 2005-08-01 2012-12-04 Allergan, Inc. Botulinum toxin compositions
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
EP3611266B1 (en) 2005-08-23 2022-11-09 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
WO2007026353A2 (en) 2005-08-29 2007-03-08 Technion Research & Development Foundation Ltd. Media for culturing stem cells
TW200732347A (en) 2005-10-06 2007-09-01 Trophogen Inc VEGF analogs and methods of use
BRPI0619794B8 (pt) 2005-12-13 2022-06-14 Univ Kyoto Uso de um fator de reprogramação, agente para a preparação de uma célula-tronco pluripotente induzida a partir de uma célula somática e métodos para preparar uma célula- tronco pluripotente induzida método e para preparar uma célula somática e uso de células-tronco pluripotentes induzidas
US8129187B2 (en) 2005-12-13 2012-03-06 Kyoto University Somatic cell reprogramming by retroviral vectors encoding Oct3/4. Klf4, c-Myc and Sox2
US8278104B2 (en) 2005-12-13 2012-10-02 Kyoto University Induced pluripotent stem cells produced with Oct3/4, Klf4 and Sox2
CA2634329A1 (en) 2005-12-22 2007-07-19 Csl Behring Gmbh Octanoate-reduced human albumin
US7658728B2 (en) 2006-01-10 2010-02-09 Yuzhakov Vadim V Microneedle array, patch, and applicator for transdermal drug delivery
DE102006051516A1 (de) 2006-10-31 2008-05-08 Curevac Gmbh (Basen-)modifizierte RNA zur Expressionssteigerung eines Proteins
GB0623635D0 (en) 2006-11-27 2007-01-03 Stem Cell Sciences Uk Ltd Pluripotent cell growth media
US7785301B2 (en) 2006-11-28 2010-08-31 Vadim V Yuzhakov Tissue conforming microneedle array and patch for transdermal drug delivery or biological fluid collection
EP2099496A2 (en) 2006-12-08 2009-09-16 Massachusetts Institute of Technology Delivery of nanoparticles and/or agents to cells
CN101200758A (zh) 2006-12-15 2008-06-18 华中科技大学 一种检测col7a1基因突变的方法及其用途
US10829733B2 (en) 2007-01-04 2020-11-10 Biolamina Ab Composition and method for enabling proliferation of pluripotent human stem cells
JP2010515464A (ja) 2007-01-11 2010-05-13 イエール・ユニバーシテイ Hiv細胞表面受容体の標的化不活性化のための組成物および方法
US9249423B2 (en) 2007-02-02 2016-02-02 Yale University Method of de-differentiating and re-differentiating somatic cells using RNA
US8859229B2 (en) 2007-02-02 2014-10-14 Yale University Transient transfection with RNA
DK3399025T3 (da) 2007-03-23 2025-06-16 Wisconsin Alumni Res Found Omprogrammering af somatiske celler
EP2626416A3 (en) 2007-04-07 2013-12-18 The Whitehead Institute for Biomedical Research Reprogramming of somatic cells
EP2167539A2 (en) * 2007-06-15 2010-03-31 Charite-Universitätsmedizin Berlin Methods for producing glycosylated human alpha-1 antitrypsin (a1at) in mammalian host cells
JP2008307007A (ja) 2007-06-15 2008-12-25 Bayer Schering Pharma Ag 出生後のヒト組織由来未分化幹細胞から誘導したヒト多能性幹細胞
JP5558097B2 (ja) 2007-12-10 2014-07-23 国立大学法人京都大学 効率的な核初期化方法
EP2234929A1 (en) 2007-12-14 2010-10-06 DSM IP Assets B.V. Sol-gel process with a protected catalyst
EP2072618A1 (en) 2007-12-14 2009-06-24 Johannes Gutenberg-Universität Mainz Use of RNA for reprogramming somatic cells
EP2222696A1 (en) 2007-12-21 2010-09-01 Stryker Corporation Bmp mutants with decreased susceptibility to noggin
SG188904A1 (en) 2008-03-17 2013-04-30 Scripps Research Inst Combined chemical and genetic approaches for generation of induced pluripotent stem cells
US20110045001A1 (en) 2008-03-28 2011-02-24 Biontex Laboratories Gmbh Transfection results of non-viral gene delivery systems by influencing of the innate immune system
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
JP2011160661A (ja) 2008-06-02 2011-08-25 Kyowa Hakko Kirin Co Ltd 血球細胞の初期化法
EP3279314A1 (en) 2008-06-04 2018-02-07 Cellular Dynamics International, Inc. Methods for the production of ips cells using non-viral approach
GB2460552B (en) 2008-06-05 2011-09-07 Iti Scotland Ltd Stem cell culture media and methods
US8669048B2 (en) 2008-06-24 2014-03-11 Parkinson's Institute Pluripotent cell lines and methods of use thereof
US20100184033A1 (en) 2008-07-16 2010-07-22 West Michael D Methods to accelerate the isolation of novel cell strains from pluripotent stem cells and cells obtained thereby
EP2151248A1 (en) 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
WO2010022194A2 (en) 2008-08-20 2010-02-25 Virxsys Corporation Compositions and methods for generation of pluripotent stem cells
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
CA2741090C (en) 2008-10-24 2018-10-16 Wisconsin Alumni Research Foundation Pluripotent stem cells obtained by non-viral reprogramming
JP2012509106A (ja) 2008-11-18 2012-04-19 スリーエム イノベイティブ プロパティズ カンパニー 中空のマイクロニードルアレイ及び方法
EP2881461A1 (en) 2008-11-21 2015-06-10 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Reprogramming cells toward a pluripotent state
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
EP2412800A1 (en) 2010-07-29 2012-02-01 Koninklijke Nederlandse Akademie van Wetenschappen Liver organoid, uses thereof and culture method for obtaining them
WO2010093655A2 (en) 2009-02-10 2010-08-19 University Of Dayton Enhanced method for producing stem-like cells from somatic cells
US10894944B2 (en) 2009-04-10 2021-01-19 Monash University Cell culture media
US20100273220A1 (en) 2009-04-22 2010-10-28 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long rna molecules
US10837020B2 (en) 2009-04-22 2020-11-17 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long RNA molecules
US20100272695A1 (en) 2009-04-22 2010-10-28 Alan Agulnick Cell compositions derived from dedifferentiated reprogrammed cells
EP2251437B1 (en) 2009-05-13 2013-12-04 Hannelore Breitenbach-Koller Method for identifying compounds that control the translational activity of ribosomal proteins in differential mRNA expression
US8496941B2 (en) 2009-06-03 2013-07-30 National Institute Of Advanced Industrial Science And Technology Vectors for generating pluripotent stem cells and methods of producing pluripotent stem cells using the same
CA2765700C (en) 2009-06-16 2021-01-05 Opko Curna, Llc Treatment of collagen gene related diseases by inhibition of natural antisense transcript to a collagen gene
BR112012002291A2 (pt) 2009-07-31 2016-11-29 Ethris Gmbh "polirribonucleotídeo com uma sequência que codifica uma proteína ou fragmento de proteína, implante, e, processo para a seleção de sequências de nucleotídeos"
ES2539487T3 (es) 2009-11-04 2015-07-01 Cellular Dynamics International, Inc. Reprogramación episómica con compuestos químicos
EP2499239A1 (en) 2009-11-11 2012-09-19 Sanford-Burnham Medical Research Institute METHOD FOR GENERATION AND REGULATION OF iPS CELLS AND COMPOSITIONS THEREOF
US20130189741A1 (en) 2009-12-07 2013-07-25 Cellscript, Inc. Compositions and methods for reprogramming mammalian cells
KR20240136456A (ko) 2009-12-07 2024-09-13 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 세포 리프로그래밍을 위한 정제된 변형 rna를 포함하는 rna 제제
HUE041436T2 (hu) 2009-12-10 2019-05-28 Univ Minnesota Tal-effektor-közvetített DNS-módosítás
US8557972B2 (en) 2009-12-21 2013-10-15 University Of Washington Through Its Center For Commercialization Compositions and methods for transfection of RNA and controlled stabilization of transfected RNA
CA2788635A1 (en) 2010-02-01 2011-08-04 The Board Of Trustees Of The Leland Stanford Junior University Enhanced efficiency of induced pluripotent stem cell generation
US20140194482A1 (en) 2010-02-02 2014-07-10 Scioderm, Inc. Compositions and methods of treatment of inflammatory skin conditions using allantoin
JPWO2011102532A1 (ja) 2010-02-16 2013-06-17 国立大学法人九州大学 誘導肝細胞
WO2011110886A1 (en) 2010-03-09 2011-09-15 Biolamina Ab Composition and method for enabling proliferation of pluripotent human stem cells
CA2796464C (en) 2010-04-16 2021-08-03 Immune Disease Institute, Inc. Sustained polypeptide expression from synthetic, modified rnas and uses thereof
CN102234627B (zh) 2010-04-30 2015-06-03 中国科学院广州生物医药与健康研究院 一种培养基添加剂及其应用
WO2011140397A2 (en) 2010-05-05 2011-11-10 The Regents Of The University Of California Office Of The President Stem cell defined media for xeno-free and feeder free conditions and uses thereof
US8048675B1 (en) 2010-05-12 2011-11-01 Ipierian, Inc. Integration-free human induced pluripotent stem cells from blood
US20130145487A1 (en) 2010-05-12 2013-06-06 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
JP6208580B2 (ja) 2010-05-17 2017-10-04 サンガモ セラピューティクス, インコーポレイテッド 新規のdna結合タンパク質及びその使用
EP2392208B1 (en) 2010-06-07 2016-05-04 Helmholtz Zentrum München Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Fusion proteins comprising a DNA-binding domain of a Tal effector protein and a non-specific cleavage domain of a restriction nuclease and their use
AU2011265733B2 (en) 2010-06-14 2014-04-17 Iowa State University Research Foundation, Inc. Nuclease activity of TAL effector and Foki fusion protein
EP2601288B1 (en) 2010-08-05 2016-04-06 Wisconsin Alumni Research Foundation Simplified basic media for human pluripotent cell culture
WO2012019168A2 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
WO2012021632A2 (en) 2010-08-10 2012-02-16 The Johns Hopkins University Generation and use of pluripotent stem cells
GB201014169D0 (en) * 2010-08-25 2010-10-06 Cambridge Entpr Ltd In vitro hepatic differentiation
JP5794588B2 (ja) 2010-09-14 2015-10-14 国立大学法人京都大学 効率的な人工多能性幹細胞の樹立方法
RU2013120302A (ru) 2010-10-01 2014-11-20 Модерна Терапьютикс, Инк. Сконструированные нуклеиновые кислоты и способы их применения
WO2012048213A1 (en) 2010-10-08 2012-04-12 Regents Of The University Of Minnesota A method to increase gene targeting frequency
JP5888753B2 (ja) 2010-11-04 2016-03-22 国立大学法人京都大学 効率的な人工多能性幹細胞の樹立方法
WO2012094132A1 (en) 2011-01-05 2012-07-12 Sangamo Biosciences, Inc. Methods and compositions for gene correction
CA2832807A1 (en) 2011-03-07 2012-09-13 Massachusetts Institute Of Technology Methods for transfecting cells with nucleic acids
EP2691101A2 (en) 2011-03-31 2014-02-05 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
WO2012131090A1 (en) 2011-03-31 2012-10-04 Galderma Research & Development Method for treatment of xeroderma pigmentosum
EP2694660B1 (en) 2011-04-03 2018-08-08 The General Hospital Corporation Efficient protein expression in vivo using modified rna (mod-rna)
EP2694089B1 (en) 2011-04-05 2024-06-05 Cellectis New tale-protein scaffolds and uses thereof
KR102068107B1 (ko) * 2011-04-27 2020-01-20 아미리스 인코퍼레이티드 게놈 변형 방법
WO2012174224A2 (en) 2011-06-17 2012-12-20 Calando Pharmaceuticals, Inc. Methods for administering nucleic acid-based therapeutics
WO2012176015A1 (en) 2011-06-24 2012-12-27 Leo Pharma A/S Methods for treating uv-damaged skin and scc tumors and for removing tattoos with topical ingenol mebutate
US9862926B2 (en) 2011-06-27 2018-01-09 Cellscript, Llc. Inhibition of innate immune response
WO2013009825A1 (en) 2011-07-11 2013-01-17 Cellular Dynamics International, Inc. Methods for cell reprogramming and genome engineering
WO2013020064A1 (en) 2011-08-03 2013-02-07 Lotus Tissue Repair Inc. Collagen 7 and related methods
AU2012312260B2 (en) * 2011-09-21 2017-08-31 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression
RU2648950C2 (ru) 2011-10-03 2018-04-02 Модерна Терапьютикс, Инк. Модифицированные нуклеозиды, нуклеотиды и нуклеиновые кислоты и их применение
EP3279324B1 (en) 2011-10-11 2021-02-17 Institut National De La Sante Et De La Recherche Medicale (Inserm) Exon skipping therapy for dystrophic epidermolysis bullosa
BR112014008694A2 (pt) 2011-10-11 2017-06-20 Novartis Ag moléculas de ácido nucleico policistrônico recombinante
WO2013078199A2 (en) 2011-11-23 2013-05-30 Children's Medical Center Corporation Methods for enhanced in vivo delivery of synthetic, modified rnas
KR102320571B1 (ko) 2011-12-05 2021-11-02 팩터 바이오사이언스 인크. 세포를 형질감염시키는 방법들 및 생성물들
US8497124B2 (en) 2011-12-05 2013-07-30 Factor Bioscience Inc. Methods and products for reprogramming cells to a less differentiated state
WO2013090186A1 (en) 2011-12-14 2013-06-20 modeRNA Therapeutics Modified nucleic acids, and acute care uses thereof
CN104114572A (zh) 2011-12-16 2014-10-22 现代治疗公司 经修饰的核苷、核苷酸和核酸组合物
JP2015510495A (ja) 2011-12-21 2015-04-09 モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. 器官または器官移植片の生存可能性または寿命を延長する方法
EP4721752A2 (en) 2011-12-30 2026-04-08 Cellscript, Llc Herstellung und verwendung von in vitro-synthetisierter ssrna zur einführung in säugetierzellen zur induktion einer biologischen oder biochemischen wirkung
US9340784B2 (en) * 2012-03-19 2016-05-17 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
AU2013243948A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
WO2013152220A2 (en) 2012-04-04 2013-10-10 Life Technologies Corporation Tal-effector assembly platform, customized services, kits and assays
WO2013158046A1 (en) 2012-04-20 2013-10-24 Agency For Science, Technology And Research Rnai-based therapies for cardiomyopathies, muscular dystrophies and laminopathies
RU2696071C2 (ru) 2012-04-24 2019-07-30 Виселл Терапеутикс, Инк. Получение плюрипотентных клеток de novo
KR102215413B1 (ko) 2012-05-13 2021-02-16 알렐 바이오테크놀로지 앤 파마슈티칼스, 인크. 합성 메신저 rna에 의한 인간 유도 만능 줄기 세포의 피더프리 유래
US10155929B2 (en) 2012-05-13 2018-12-18 Allele Biotechnology & Pharmaceuticals, Inc. Feeder-free derivation of human-induced pluripotent stem cells with synthetic messenger RNA
US10119150B2 (en) 2012-05-13 2018-11-06 Allele Biotechnology & Pharmaceuticals, Inc. Feeder-free Derivation of human-induced pluripotent stem cells with synthetic messenger RNA
US20140031295A1 (en) 2012-07-19 2014-01-30 Lotus Tissue Repair, Inc. Recombinant C7 and Methods of Use
EP2906684B8 (en) * 2012-10-10 2020-09-02 Sangamo Therapeutics, Inc. T cell modifying compounds and uses thereof
WO2014071219A1 (en) 2012-11-01 2014-05-08 Factor Bioscience Inc. Methods and products for expressing proteins in cells
RU2718988C2 (ru) * 2013-02-22 2020-04-15 Куревак Аг Комбинация противораковой рнк-вакцины и ингибитора пути pd-1 и ее применение
ES3047667T3 (en) 2013-02-22 2025-12-04 Univ Leland Stanford Junior Methods relating to telomere extension
CN105229144A (zh) 2013-02-22 2016-01-06 细胞动力学国际有限公司 通过组合的遗传工程和化学工程经由正向编程产生肝细胞
WO2014134412A1 (en) 2013-03-01 2014-09-04 Regents Of The University Of Minnesota Talen-based gene correction
MX2015017312A (es) * 2013-06-17 2017-04-10 Broad Inst Inc Suministro y uso de composiciones, vectores y sistemas crispr-cas para la modificación dirigida y terapia hepáticas.
DK3444350T3 (da) 2013-07-03 2022-02-07 Dicerna Pharmaceuticals Inc Fremgangsmåder og sammensætninger til specifik hæmning af alpha-1 antitrypsin ved dobbeltstrenget rna
SG11201601939VA (en) 2013-09-16 2016-04-28 Agency Science Tech & Res Method
SG10201811729PA (en) 2013-12-12 2019-02-27 Life Technologies Corp Membrane-penetrating peptides to enhance transfection and compositions and methods for using same
US20150165054A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
US20150167017A1 (en) 2013-12-13 2015-06-18 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
ES2939542T3 (es) * 2014-01-31 2023-04-24 Factor Bioscience Inc Métodos y productos para la producción y la administración de ácido nucleico
ES3063961T3 (en) * 2014-02-14 2026-04-21 Cellectis Cells for immunotherapy engineered for targeting antigen present both on immune cells and pathological cells
CA2945335A1 (en) * 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
SG10201912038TA (en) * 2014-04-23 2020-02-27 Modernatx Inc Nucleic acid vaccines
WO2016069282A1 (en) * 2014-10-31 2016-05-06 The Trustees Of The University Of Pennsylvania Altering gene expression in modified t cells and uses thereof
CA3197849A1 (en) * 2014-12-29 2016-07-07 Novartis Ag Methods of making chimeric antigen receptor-expressing cells
JP7199809B2 (ja) 2015-02-13 2023-01-06 ファクター バイオサイエンス インコーポレイテッド 核酸製品及びその投与方法
CA3006618A1 (en) * 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
KR102438360B1 (ko) * 2016-03-04 2022-08-31 에디타스 메디신, 인코포레이티드 암 면역요법을 위한 crispr-cpf1-관련 방법, 조성물 및 구성성분
WO2017165862A1 (en) * 2016-03-25 2017-09-28 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
US11377637B2 (en) * 2016-04-15 2022-07-05 Memorial Sloan Kettering Cancer Center Transgenic T cell and chimeric antigen receptor T cell compositions and related methods
US10576167B2 (en) * 2016-08-17 2020-03-03 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof

Similar Documents

Publication Publication Date Title
JP2019528284A5 (https=)
Wu et al. Engineering advanced cancer therapies with synthetic biology
Shu et al. Stable RNA nanoparticles as potential new generation drugs for cancer therapy
JP2017510542A5 (https=)
US11180730B2 (en) Compositions and methods for evaluating and modulating immune responses by detecting and targeting GATA3
WO2020074001A1 (en) Methods and Compositions for Editing RNAs
EP3368689B1 (en) Composition for modulating immune responses by use of immune cell gene signature
US20180100201A1 (en) Tumor and microenvironment gene expression, compositions of matter and methods of use thereof
ES3040945T3 (en) Materials and methods for treatment of duchenne muscular dystrophy
JP2022526455A (ja) Rnaを編集する方法および組成物
Lau et al. CRISPR-based strategies for targeted transgene knock-in and gene correction
Lu et al. Multifunctional DNA origami nanoplatforms for drug delivery
US20190106678A1 (en) Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1
Bode et al. Exploiting single-cell tools in gene and cell therapy
Sambasivan et al. Embryonic founders of adult muscle stem cells are primed by the determination gene Mrf4
FI3599251T3 (fi) Cd20-antigeeniin kohdentuvan kimeerisen antigeenireseptorin rakentaminen ja sillä muunnettujen t-solujen aktiivisuuden tunnistaminen
US20220033792A1 (en) Methods and compositions for cellular reprogramming
JP2017537627A5 (https=)
JP2017523777A5 (https=)
Liu et al. Viral nanoparticle-encapsidated enzyme and restructured DNA for cell delivery and gene expression
US20260078383A1 (en) Rna nanostructures and methods of making and using rna nanostructures
Perera et al. Peptide nucleic acids and their role in gene regulation and editing
Yan et al. Advances in RNA-based cancer therapeutics: pre-clinical and clinical implications
CN109963944A (zh) 体外敲除T细胞中靶基因的方法以及该方法中使用的crRNA
Shamjetsabam et al. CRISPR/Cas9: an overview of recent developments and applications in cancer research