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 JP2022168830A priority Critical patent/JP2023011697A/ja
Priority to JP2022168829A priority patent/JP2023011696A/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
JP2022168830A JP2023011697A (ja) 2016-08-17 2022-10-21 核酸産物およびその投与方法
JP2022168829A JP2023011696A (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 (enExample) 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
JP2022168829A Pending JP2023011696A (ja) 2016-08-17 2022-10-21 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

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2022168829A Pending JP2023011696A (ja) 2016-08-17 2022-10-21 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

Country Status (8)

Country Link
US (12) US10576167B2 (enExample)
EP (1) EP3500585A4 (enExample)
JP (3) JP2019528284A (enExample)
CN (2) CN116115629A (enExample)
AU (2) AU2017312113B2 (enExample)
CA (1) CA3033788A1 (enExample)
IL (2) IL308824A (enExample)
WO (1) WO2018035377A1 (enExample)

Families Citing this family (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103893747B (zh) * 2007-11-01 2016-01-06 阿肯色大学评议会 增强针对艾美球虫属的免疫反应的组合物和方法
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
WO2018035377A1 (en) * 2016-08-17 2018-02-22 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
EP3596108A4 (en) 2017-03-15 2020-12-23 Pandion Operations, Inc. TARGETED IMMUNOTOLERANCE
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
CN111010866A (zh) 2017-05-24 2020-04-14 潘迪恩治疗公司 靶向免疫耐受性
AU2018278315B2 (en) 2017-05-31 2024-01-18 Ultragenyx Pharmaceutical Inc. Therapeutics for glycogen storage disease type III
JP7423522B2 (ja) 2017-11-22 2024-01-29 モダーナティエックス・インコーポレイテッド 尿素サイクル異常症の治療のためのオルニチントランスカルバミラーゼをコードするポリヌクレオチド
US10174091B1 (en) 2017-12-06 2019-01-08 Pandion Therapeutics, Inc. IL-2 muteins
US10946068B2 (en) 2017-12-06 2021-03-16 Pandion Operations, Inc. IL-2 muteins and uses thereof
USRE50550E1 (en) 2017-12-06 2025-08-26 Pandion Operations, Inc. IL-2 muteins and uses thereof
WO2019191341A1 (en) * 2018-03-27 2019-10-03 Factor Bioscience Inc. Nucleic acid-based therapeutics
CN112601816B (zh) * 2018-05-11 2024-12-17 比姆医疗股份有限公司 使用可编程碱基编辑器系统遏止病原性突变的方法
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
HUE064076T2 (hu) 2018-12-06 2024-02-28 Arcturus Therapeutics Inc Készítmények és módszerek az ornitin-transzkarbamiláz hiányának kezelésére
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
CN114679909A (zh) 2019-05-20 2022-06-28 潘迪恩运营公司 MAdCAM靶向的免疫耐受
US20220259617A1 (en) 2019-06-13 2022-08-18 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
AU2020301036A1 (en) 2019-07-03 2022-02-17 Factor Bioscience Inc. Cationic lipids and uses thereof
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
CN214973877U (zh) 2019-08-09 2021-12-03 胡桃钳医疗公司 用于形成治疗性多核苷酸的微流体设备及微流体路径装置
BR112022003617A2 (pt) * 2019-09-01 2022-09-13 Exuma Biotech Corp Métodos e composições para a modificação e distribuição de linfócitos
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
JP7451705B2 (ja) * 2019-11-26 2024-03-18 コリア リサーチ インスティテュート オブ バイオサイエンス アンド バイオテクノロジー タンパク質発現用mRNA構造体及びその用途
EP4107187A4 (en) 2020-02-21 2024-07-03 Pandion Operations, Inc. TISSUE-TARGETED IMMUNOTOLERANCE WITH A CD39 EFFECTOR
IL295888A (en) * 2020-02-24 2022-10-01 Us Health Nk cells or t cells expressing hematopoietic growth factor receptors and use for treating cancer
EP4110465A4 (en) * 2020-02-24 2024-06-12 The Board Of Regents Of The University Of Texas System MOLECULAR BIOMARKERS AND TARGETS FOR FUCHS ENDOTHELIAL CORNEAL DYSTROPHY AND GLAUCOMA
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
US12351834B2 (en) 2020-03-03 2025-07-08 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
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
IL300001A (en) 2020-07-24 2023-03-01 Massachusetts Gen Hospital 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
US20240000891A1 (en) * 2020-12-08 2024-01-04 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
IL307433A (en) * 2021-05-03 2023-12-01 Ageronix SA Alpha-1-antitrypsin (aat) in the treatment and/or prevention of neurological disorders
CA3214286A1 (en) 2021-05-07 2022-11-10 Nikhil Dhar Modified ribonucleic acids and uses thereof
US20250099561A1 (en) * 2022-01-21 2025-03-27 Luis Hector BARBEITO ERBA Active immunization for reducing osteoarthritic, neuropathic, and cancer pain
EP4526459A2 (en) 2022-05-17 2025-03-26 Nvelop Therapeutics, Inc. Compositions and methods for efficient in vivo delivery
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
WO2025155773A1 (en) * 2024-01-19 2025-07-24 Gene Company (Pty)Ltd. Telomerase upregulating polynucleotide 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

Family Cites Families (184)

* 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
US5849686A (en) 1991-03-11 1998-12-15 Creative Biomolecules, Inc. Morphogen-induced liver 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
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
US5656593A (en) 1991-03-11 1997-08-12 Creative Biomolecules, Inc. Morphogen induced periodontal tissue regeneration
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
EP0904093A4 (en) 1996-03-22 2004-11-17 Gen Hospital Corp THE ADMINISTRATION OF GROWTH FACTOR POLYPEPTIDES AFTER AN ISCHEMICAL OF THE CENTRAL NERVOUS SYSTEM OR A TRAUMA
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
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
CA2263705C (en) 1996-08-19 2007-12-04 Nancy Smyth-Templeton Novel sandwich liposome complexes comprising a biologically active agent
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
EP1021549A2 (en) 1997-09-19 2000-07-26 Sequitur, Inc. 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
DE60044084D1 (de) 1999-06-04 2010-05-12 Georgia Tech Res Inst Vorrichtungen zur vergrösserten penetration von mikronadeln in biologischen hautschichten
US7621606B2 (en) 2001-08-27 2009-11-24 Advanced Cell Technology, Inc. Trans-differentiation and re-differentiation of somatic cells and production of cells for cell therapies
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
KR100894767B1 (ko) 2000-09-26 2009-04-24 이데라 파마슈티칼즈, 인코포레이티드 화학적인 위치 변화에 의해 면역자극 올리고누클레오티드유사체의 면역자극 활성을 조절하는 방법
US7189759B2 (en) 2001-05-23 2007-03-13 Medicis Pharmaceutical Corporation Compositions for the treatment of pigmentation disorders and methods for their manufacture
EP1437403A4 (en) 2001-09-21 2004-10-27 Japan Science & Tech Corp METHOD FOR PATTERNING REPROGRAMMING FACTOR, METHOD OF PATTERNED REPROGRAMMING FACTOR, METHOD FOR USE OF REPROGRAMMING FACTOR, METHOD FOR DIFFERENTIATING AND SIFE-INGENATING FAN
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
US7541344B2 (en) 2003-06-03 2009-06-02 Eli Lilly And Company Modulation of survivin expression
EP1691780A2 (en) 2003-11-21 2006-08-23 Alza Corporation Gene delivery mediated by liposome-dna complex with cleavable peg surface modification
US7682828B2 (en) 2003-11-26 2010-03-23 Whitehead Institute For Biomedical Research Methods for reprogramming somatic cells
BRPI0514641A (pt) 2004-09-08 2008-06-17 Wisconsin Alumni Res Found cultivação das células tronco embriÈnica humana
EP1791948B1 (en) 2004-09-08 2012-03-14 Wisconsin Alumni Research Foundation Medium and culture of embryonic stem cells
US20090202638A2 (en) 2004-10-27 2009-08-13 New York Society For The Ruptured And Crippled Maintaining The Hospital For Special Surgery Bmp gene and fusion protein
AU2006208199B2 (en) 2005-01-25 2012-06-07 Cell Therapeutics, Inc. Conjugates of biologically active proteins having a modified in vivo half-life
US7696318B2 (en) 2005-07-13 2010-04-13 Novo Nordisk Health Care Ag Host cell protein knock-out cells for production of therapeutic proteins
SG10201508995QA (en) 2005-07-26 2015-11-27 Sangamo Biosciences 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
LT2578685T (lt) 2005-08-23 2019-06-10 The Trustees Of The University Of Pennsylvania Rnr, apimančios modifikuotus nukleozidus ir jų panaudojimo būdai
US8476070B2 (en) 2005-08-29 2013-07-02 Technion Research & Development Foundation Limited Media for culturing stem cells
TW200732347A (en) 2005-10-06 2007-09-01 Trophogen Inc VEGF analogs and methods of use
WO2007069666A1 (ja) 2005-12-13 2007-06-21 Kyoto University 核初期化因子
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
US20100168000A1 (en) 2005-12-22 2010-07-01 Peter Kiessling 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
CA2671850A1 (en) 2006-12-08 2008-06-19 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
EP2099911A2 (en) 2007-01-11 2009-09-16 Yale University Compositions and methods for targeted inactivation of hiv cell surface receptors
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
SG193652A1 (en) 2007-03-23 2013-10-30 Wisconsin Alumni Res Found Somatic cell reprogramming
EP3878949B1 (en) 2007-04-07 2025-06-04 Whitehead Institute for Biomedical Research Reprogramming of somatic 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
WO2008151845A2 (en) * 2007-06-15 2008-12-18 Charite - Universitätsmedizin Berlin Methods for producing glycosylated human alpha-1 antitrypsin (a1at) in mammalian host cells
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
US20110021335A1 (en) 2007-12-14 2011-01-27 Pascal Jozef Paul Buskens 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
CN102027105B (zh) 2008-03-17 2015-03-11 斯克里普斯研究所 用于产生诱导的多能干细胞的组合化学遗传方法
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
DK2297307T3 (en) 2008-06-04 2016-07-25 Cellular Dynamics Int Inc PROCEDURES FOR THE MANUFACTURE OF IPS CELLS USING NON-VIRAL METHODS
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
EP2326711A2 (en) 2008-08-20 2011-06-01 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
CN105802917A (zh) 2008-10-24 2016-07-27 威斯康星校友研究基金会 通过非病毒重编程获得的多潜能干细胞
CN105999538A (zh) 2008-11-18 2016-10-12 3M创新有限公司 空心微针阵列和方法
ES2561949T3 (es) 2008-11-21 2016-03-01 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Reprogramación de células hacia un estado pluripotente
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
EP3904505A1 (en) 2009-04-22 2021-11-03 Viacyte, Inc. Cell compositions derived from dedifferentiated reprogrammed cells
US10837020B2 (en) * 2009-04-22 2020-11-17 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long RNA molecules
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
WO2010148050A2 (en) 2009-06-16 2010-12-23 Curna, Inc. Treatment of collagen gene related diseases by inhibition of natural antisense transcript to a collagen gene
KR20140119197A (ko) 2009-07-31 2014-10-08 에트리스 게엠베하 단백질 발현을 위한 비변형된 뉴클레오티드 및 변형된 뉴클레오티드의 조합을 가진 rna
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
US9295697B2 (en) 2009-11-04 2016-03-29 Cellular Dynamics International, Inc. Episomal reprogramming with chemicals
CN102712904A (zh) 2009-11-11 2012-10-03 桑福德—伯恩哈姆医学研究协会 用于产生和调节iPS细胞的方法及其组合物
US20130189741A1 (en) 2009-12-07 2013-07-25 Cellscript, Inc. Compositions and methods for reprogramming mammalian cells
US8808982B2 (en) 2009-12-07 2014-08-19 Cellscript, Llc Compositions and methods for reprogramming eukaryotic cells
US8586363B2 (en) 2009-12-10 2013-11-19 Regents Of The University Of Minnesota TAL effector-mediated DNA modification
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
CA3122219A1 (en) 2010-04-16 2011-10-20 The Children's Hospital Corporation 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
US9725695B2 (en) 2010-05-05 2017-08-08 The Regents Of The University Of California 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
CA2799095A1 (en) 2010-05-12 2011-11-17 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
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
EP2580331A4 (en) 2010-06-14 2013-11-27 Univ Iowa State Res Found Inc NUCLEASE ACTIVITY OF THE TAL EFFECTOR AND FUSION PROTEIN FOKI
CA2807418C (en) 2010-08-05 2017-04-04 Wisconsin Alumni Research Foundation Simplified basic media for human pluripotent cell culture
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
US20140127814A1 (en) 2010-08-10 2014-05-08 Srinivasan Chandrasegaran Generation and use of pluripotent stem cells
GB201014169D0 (en) * 2010-08-25 2010-10-06 Cambridge Entpr Ltd In vitro hepatic differentiation
US9447408B2 (en) 2010-09-14 2016-09-20 Kyoto University Method of efficiently establishing induced pluripotent stem cells
EP4108671B1 (en) 2010-10-01 2024-11-20 ModernaTX, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
WO2012048213A1 (en) 2010-10-08 2012-04-12 Regents Of The University Of Minnesota A method to increase gene targeting frequency
US9637732B2 (en) 2010-11-04 2017-05-02 Kyoto University Method of efficiently establishing induced pluripotent stem cells
WO2012094132A1 (en) 2011-01-05 2012-07-12 Sangamo Biosciences, Inc. Methods and compositions for gene correction
AU2012225497A1 (en) 2011-03-07 2013-10-24 Massachusetts Institute Of Technology Methods for transfecting cells with nucleic acids
US8710200B2 (en) 2011-03-31 2014-04-29 Moderna Therapeutics, Inc. Engineered nucleic acids encoding a modified erythropoietin and their expression
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)
WO2012138927A2 (en) 2011-04-05 2012-10-11 Philippe Duchateau Method for the generation of compact tale-nucleases and uses thereof
PT2702160T (pt) * 2011-04-27 2020-07-30 Amyris Inc Métodos para modificação genómica
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
EP2732029B1 (en) 2011-07-11 2019-01-16 FUJIFILM Cellular Dynamics, Inc. Methods for cell reprogramming and genome engineering
JP2014524241A (ja) 2011-08-03 2014-09-22 ロータス ティシュー リペア インコーポレイテッド コラーゲン7及び関連する方法
CA3186126A1 (en) * 2011-09-21 2013-03-28 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
ES2911677T3 (es) 2011-10-03 2022-05-20 Modernatx Inc Nucleósidos, nucleótidos y ácidos nucleicos modificados, y sus usos
US9340783B2 (en) 2011-10-11 2016-05-17 Inserm (Institut National De La Sante Et De La Recherche Medicale Exon skipping therapy for dystrophic epidermolysis bullosa
CN104105504A (zh) 2011-10-11 2014-10-15 诺华股份有限公司 重组多顺反子核酸分子
WO2013078199A2 (en) 2011-11-23 2013-05-30 Children's Medical Center Corporation Methods for enhanced in vivo delivery of synthetic, modified rnas
US8497124B2 (en) 2011-12-05 2013-07-30 Factor Bioscience Inc. Methods and products for reprogramming cells to a less differentiated state
KR20250035015A (ko) 2011-12-05 2025-03-11 팩터 바이오사이언스 인크. 세포를 형질감염시키는 방법들 및 생성물들
WO2013090186A1 (en) 2011-12-14 2013-06-20 modeRNA Therapeutics Modified nucleic acids, and acute care uses thereof
MX2014007233A (es) 2011-12-16 2015-02-04 Moderna Therapeutics Inc Composiciones de nucleosidos, nucleotidos y acidos nucleicos modificados.
EP2793906A4 (en) 2011-12-21 2016-01-13 Moderna Therapeutics Inc METHOD FOR INCREASING THE LIFE-LIFE OR DURABILITY OF AN ORGAN OR ORGAN EXPLOITATE
DK3421601T3 (da) 2011-12-30 2020-02-24 Cellscript Llc Fremstilling og anvendelse af in-vitro-syntetiseret ssRNA til indføring i pattedyrceller med henblik på at inducere en biologisk eller biokemisk effekt
WO2013142514A1 (en) * 2012-03-19 2013-09-26 Isis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
WO2013151663A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
US20130274129A1 (en) 2012-04-04 2013-10-17 Geneart Ag Tal-effector assembly platform, customized services, kits and assays
SG11201406787TA (en) 2012-04-20 2014-12-30 Agency Science Tech & Res Rnai-based therapies for cardiomyopathies, muscular dystrophies and laminopathies
CN104718283A (zh) 2012-04-24 2015-06-17 布里格姆及妇女医院股份有限公司 从头生成多能细胞
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
ES2951822T3 (es) 2012-05-13 2023-10-25 Allele Biotechnology & Pharmaceuticals Inc Derivación sin alimentadores de células madre pluripotentes inducidas humanas con ARN mensajero sintético
WO2014015314A1 (en) 2012-07-19 2014-01-23 University Of Southern California Recombinant c7 and methods of use
WO2014059173A2 (en) * 2012-10-10 2014-04-17 Sangamo Biosciences, 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
SI2959005T1 (sl) 2013-02-22 2022-01-31 The Board Of Trustees Of The Leland Stanford Junior University Medicinska uporaba v zvezi z ekstenzijo telomera
ES2739913T3 (es) 2013-02-22 2020-02-04 Curevac Ag Combinación de vacunación e inhibición de la ruta PD-1
AU2014218807A1 (en) 2013-02-22 2015-09-03 Cellular Dynamics International, Inc. Hepatocyte production via forward programming by combined genetic and chemical engineering
ES2750550T3 (es) 2013-03-01 2020-03-26 Univ Minnesota Corrección de gen a base de TALEN
SG11201510284XA (en) * 2013-06-17 2016-01-28 Broad Inst Inc Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
EP3444350B1 (en) 2013-07-03 2021-12-01 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna
WO2015038075A1 (en) 2013-09-16 2015-03-19 Agency For Science, Technology And Research Method
US11053481B2 (en) * 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
CA2933561A1 (en) 2013-12-12 2015-06-18 Life Technologies Corporation Membrane-penetrating peptides to enhanced transfection and compositions and methods for using same
EP3053585A1 (en) 2013-12-13 2016-08-10 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
AU2015210769B2 (en) * 2014-01-31 2019-02-07 Factor Bioscience Inc. Methods and products for nucleic acid production and delivery
PT3105317T (pt) * 2014-02-14 2019-02-27 Cellectis Células para imunoterapia manipuladas para atuar sobre antigénios presentes tanto em células imunitárias como em células patológicas
KR20250102123A (ko) * 2014-04-18 2025-07-04 에디타스 메디신, 인코포레이티드 암 면역요법을 위한 crispr-cas-관련 방법, 조성물 및 구성성분
PT3134131T (pt) * 2014-04-23 2022-03-24 Modernatx Inc Vacinas de ácidos nucleicos
KR20250075716A (ko) * 2014-12-29 2025-05-28 노파르티스 아게 키메라 항원 수용체-발현 세포를 제조하는 방법
EP4406585A3 (en) 2015-02-13 2024-10-23 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
EP3967758A1 (en) * 2015-12-01 2022-03-16 CRISPR Therapeutics AG Materials and methods for treatment of alpha-1 antitrypsin deficiency
IL291971B2 (en) * 2016-03-04 2025-04-01 Editas Medicine Inc CRISPR–CPF1 related methods, compositions and components for cancer immunotherapy
US11512311B2 (en) * 2016-03-25 2022-11-29 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency
WO2018035377A1 (en) * 2016-08-17 2018-02-22 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof

Similar Documents

Publication Publication Date Title
JP2019528284A5 (enExample)
JP7698828B2 (ja) Rnaを編集する方法および組成物
CN113631708B (zh) 编辑rna的方法和组合物
Wu et al. Engineering advanced cancer therapies with synthetic biology
RU2020104969A (ru) Способы и продукты для получения и доставки нуклеиновых кислот
JP2017510542A5 (enExample)
Falzarano et al. Duchenne muscular dystrophy: from diagnosis to therapy
US11186825B2 (en) Compositions and methods for evaluating and modulating immune responses by detecting and targeting POU2AF1
US11180730B2 (en) Compositions and methods for evaluating and modulating immune responses by detecting and targeting GATA3
US20180100201A1 (en) Tumor and microenvironment gene expression, compositions of matter and methods of use thereof
US20190100801A1 (en) Compositions and methods for evaluating and modulating immune responses by use of immune cell gene signatures
Sambasivan et al. Embryonic founders of adult muscle stem cells are primed by the determination gene Mrf4
RU2016123086A (ru) Способы и композиции для лечения болезни хантингтона
JP2016116520A5 (enExample)
JP2017537627A5 (enExample)
JP2017535258A5 (enExample)
JP2017523777A5 (enExample)
JP2009159971A5 (enExample)
JP2012050449A5 (enExample)
Liu et al. Viral nanoparticle-encapsidated enzyme and restructured DNA for cell delivery and gene expression
Perera et al. Peptide nucleic acids and their role in gene regulation and editing
US20180119122A1 (en) Methods and compositions for cellular reprogramming
Chenna et al. A simple cytosine-to-G-clamp nucleobase substitution enables chiral γ-PNAs to invade mixed-sequence double helical B-form DNA
Wang et al. CRISPR applications in cancer diagnosis and treatment
Meng et al. Optimization of CRISPR–Cas system for clinical cancer therapy