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
JP2022168829A Division JP2023011696A (ja) 2016-08-17 2022-10-21 核酸産物およびその投与方法
JP2022168830A Division JP2023011697A (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 (OSRAM) 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 (OSRAM)
EP (1) EP3500585A4 (OSRAM)
JP (3) JP2019528284A (OSRAM)
CN (2) CN116115629A (OSRAM)
AU (2) AU2017312113B2 (OSRAM)
CA (1) CA3033788A1 (OSRAM)
IL (2) IL308824A (OSRAM)
WO (1) WO2018035377A1 (OSRAM)

Families Citing this family (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT2214701T (pt) * 2007-11-01 2016-11-02 Univ Guelph Composições e métodos de potenciar respostas imunes a eimeria
KR102315098B1 (ko) 2012-11-01 2021-10-21 팩터 바이오사이언스 인크. 세포에서 단백질을 발현시키는 방법들과 생성물들
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
US11111505B2 (en) 2016-03-19 2021-09-07 Exuma Biotech, Corp. Methods and compositions for transducing 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
CN111010866A (zh) 2017-05-24 2020-04-14 潘迪恩治疗公司 靶向免疫耐受性
JP7284101B2 (ja) 2017-05-31 2023-05-30 ウルトラジェニクス ファーマシューティカル インク． 糖原病ｉｉｉ型のための治療薬
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
USRE50550E1 (en) 2017-12-06 2025-08-26 Pandion Operations, Inc. IL-2 muteins and uses thereof
US10174092B1 (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
EP3775202A4 (en) * 2018-03-27 2022-04-06 Factor Bioscience Inc. NUCLEIC ACID BASED THERAPEUTICS
EP3790964A4 (en) * 2018-05-11 2022-06-08 Beam Therapeutics, Inc. METHODS FOR REMOVAL OF PATHOGENIC MUTATIONS USING PROGRAMMABLE BASE EDITOR SYSTEMS
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
US11685906B2 (en) 2018-12-06 2023-06-27 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
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
US11739146B2 (en) 2019-05-20 2023-08-29 Pandion Operations, Inc. MAdCAM targeted immunotolerance
JP2022536364A (ja) 2019-06-13 2022-08-15 ザ ジェネラル ホスピタル コーポレイション 操作されたヒト内在性ウイルス様粒子および細胞への送達のためのその使用方法
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
CN114450265B (zh) 2019-07-03 2024-12-24 菲克特生物科学股份有限公司 阳离子脂质及其用途
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
MX2022001468A (es) 2019-08-09 2022-07-19 Nutcracker Therapeutics Inc Metodos y aparatos de fabricación para remover material de una composición terapeutica.
KR20220070449A (ko) * 2019-09-01 2022-05-31 엑수마 바이오테크, 코포레이션 림프구의 변형 및 전달을 위한 방법 및 조성물
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
WO2021107647A1 (ko) * 2019-11-26 2021-06-03 한국생명공학연구원 단백질 발현용 mrna 구조체 및 이의 용도
US11981715B2 (en) 2020-02-21 2024-05-14 Pandion Operations, Inc. Tissue targeted immunotolerance with a CD39 effector
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
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
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
CA3169889A1 (en) 2020-03-03 2021-09-10 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
EP4149252A4 (en) * 2020-05-12 2024-10-02 Factor Bioscience Inc. GENETICALLY ENGINEERED 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
WO2022020332A1 (en) 2020-07-21 2022-01-27 Chembeau LLC Diester cosmetic formulations and uses thereof
WO2022020800A2 (en) 2020-07-24 2022-01-27 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
KR20240004531A (ko) * 2021-05-03 2024-01-11 아제로닉스 에스에이 신경 장애의 치료 및/또는 예방을 위한 알파-1-항트립신(aat)
CA3214286A1 (en) 2021-05-07 2022-11-10 Nikhil Dhar Modified ribonucleic acids and uses thereof
CA3243118A1 (en) * 2022-01-21 2023-07-27 Pralonir S.A.S. ACTIVE IMMUNIZATION TO REDUCE ARTHRITIS, 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
CN116585342B (zh) * 2023-05-23 2024-08-16 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
US5656593A (en) 1991-03-11 1997-08-12 Creative Biomolecules, Inc. Morphogen induced periodontal tissue regeneration
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
US6800603B2 (en) 1991-03-11 2004-10-05 Curis, Inc. Morphogen-induced neural cell adhesion
US5849686A (en) 1991-03-11 1998-12-15 Creative Biomolecules, Inc. Morphogen-induced liver regeneration
US6506729B1 (en) 1991-03-11 2003-01-14 Curis, Inc. Methods and compositions for the treatment and prevention of Parkinson's disease
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
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
ATE209902T1 (de) 1996-08-19 2001-12-15 Us Gov Health & Human Serv Neue liposomkomplexe mit erhöhter systemischer verabreichung
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
AU5734998A (en) 1997-01-10 1998-08-03 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
JP2002508299A (ja) 1997-09-19 2002-03-19 セクイター， インク． センスｍＲＮＡ治療
JP4854853B2 (ja) 1998-11-12 2012-01-18 ライフ テクノロジーズ コーポレーション トランスフェクション薬剤
GB9902000D0 (en) 1999-01-30 1999-03-17 Delta Biotechnology Ltd Process
ATE462468T1 (de) 1999-06-04 2010-04-15 Georgia Tech Res Inst Vorrichtungen zur vergrösserten penetration von mikronadeln in biologischen hautschichten
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
CA2423487C (en) 2000-09-26 2015-12-15 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
WO2003018767A2 (en) 2001-08-27 2003-03-06 Advanced Cell Technology, Inc. Trans-differentiation and re-differentiation of somatic cells and production of cells for cell therapies
WO2003027277A1 (en) 2001-09-21 2003-04-03 Japan Science And Technology Corporation Method of screening reporgramming 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
JP2005506074A (ja) 2001-10-18 2005-03-03 イクシオン・バイオテクノロジー・インコーポレーテッド 肝臓の幹細胞および前駆細胞の膵臓機能細胞への転換
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
WO2005002507A2 (en) 2003-06-03 2005-01-13 Isis Pharmaceuticals, Inc. Modulation of survivin expression
JP2007512355A (ja) 2003-11-21 2007-05-17 アルザ コーポレイション 開裂性のｐｅｇで表面修飾されたリポソーム−ｄｎａ複合体で媒介される遺伝子送達
US7682828B2 (en) 2003-11-26 2010-03-23 Whitehead Institute For Biomedical Research Methods for reprogramming somatic cells
DK3196296T3 (en) 2004-09-08 2019-02-04 Wisconsin Alumini Res Foundation Cultivation of human embryonic stem cells
KR101264940B1 (ko) 2004-09-08 2013-05-15 위스콘신 얼럼나이 리서어치 화운데이션 배아 줄기 세포용 배지 및 이의 배양물
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
JP5735194B2 (ja) 2005-01-25 2015-06-17 セル セラピューティクス インコーポレーテッド 改善された生体内半減期を有する生物学的に活性なタンパク質
US7696318B2 (en) 2005-07-13 2010-04-13 Novo Nordisk Health Care Ag Host cell protein knock-out cells for production of therapeutic proteins
WO2007014275A2 (en) 2005-07-26 2007-02-01 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
ES2937245T3 (es) 2005-08-23 2023-03-27 Univ Pennsylvania ARN que contiene nucleósidos modificados y métodos de uso del mismo
EP3354723B1 (en) 2005-08-29 2023-12-13 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
US8048999B2 (en) 2005-12-13 2011-11-01 Kyoto University Nuclear reprogramming factor
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
WO2007079886A1 (de) 2005-12-22 2007-07-19 Csl Behring Gmbh Oktanoatreduziertes 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
US20090093551A1 (en) 2006-12-08 2009-04-09 Bhatia Sangeeta N Remotely triggered release from heatable surfaces
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
WO2008086529A2 (en) 2007-01-11 2008-07-17 Yale University Compositions and methods for targeted inactivation of hiv cell surface receptors
US8859229B2 (en) 2007-02-02 2014-10-14 Yale University Transient transfection with RNA
US9249423B2 (en) 2007-02-02 2016-02-02 Yale University Method of de-differentiating and re-differentiating somatic cells using RNA
EP2137296A2 (en) 2007-03-23 2009-12-30 Wisconsin Alumni Research Foundation Somatic cell reprogramming
CN101932705A (zh) 2007-04-07 2010-12-29 怀特黑德生物医学研究所 体细胞重编程
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
EP2167539A2 (en) * 2007-06-15 2010-03-31 Charite-Universitätsmedizin Berlin Methods for producing glycosylated human alpha-1 antitrypsin (a1at) in mammalian host cells
AU2008286249B2 (en) 2007-12-10 2013-10-10 Kyoto University Efficient method for nuclear reprogramming
EP2072618A1 (en) 2007-12-14 2009-06-24 Johannes Gutenberg-Universität Mainz Use of RNA for reprogramming somatic cells
WO2009077509A1 (en) 2007-12-14 2009-06-25 Dsm Ip Assets B.V. Sol-gel process with a protected catalyst
EP2222696A1 (en) 2007-12-21 2010-09-01 Stryker Corporation Bmp mutants with decreased susceptibility to noggin
SG10202103401QA (en) 2008-03-17 2021-05-28 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
JP2011522540A (ja) 2008-06-04 2011-08-04 セルラー ダイナミクス インターナショナル， インコーポレイテッド 非ウイルスアプローチを用いたｉＰＳ細胞の産生のための方法
GB2460552B (en) 2008-06-05 2011-09-07 Iti Scotland Ltd Stem cell culture media and methods
WO2010008486A2 (en) 2008-06-24 2010-01-21 Parkinsons 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
US20110263015A1 (en) 2008-08-20 2011-10-27 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
EP2356221B1 (en) 2008-10-24 2018-11-21 Wisconsin Alumni Research Foundation Pluripotent stem cells obtained by non-viral reprogramming
EP2355887B1 (en) 2008-11-18 2017-08-02 3M Innovative Properties Company Hollow microneedle array
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
EP2421563B1 (en) 2009-04-22 2017-04-12 Massachusetts Institute of Technology Innate immune suppression enables repeated delivery of long rna molecules
EP2421957B1 (en) 2009-04-22 2020-11-18 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
CN102695797B (zh) 2009-06-16 2018-05-25 库尔纳公司 通过抑制针对胶原基因的天然反义转录物来治疗胶原基因相关的疾病
EP3165234B1 (de) 2009-07-31 2019-04-03 ethris GmbH Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression
WO2011056971A2 (en) 2009-11-04 2011-05-12 Cellular Dynamics International, Inc. Episomal reprogramming with chemicals
KR20120107088A (ko) 2009-11-11 2012-09-28 샌포드-번햄 메디칼 리서치 인스티튜트 ⅰＰＳ 세포들의 생산 및 조절 방법 및 그들의 조성물들
US20130189741A1 (en) 2009-12-07 2013-07-25 Cellscript, Inc. Compositions and methods for reprogramming mammalian cells
KR101878502B1 (ko) 2009-12-07 2018-07-13 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 세포 리프로그래밍을 위한 정제된 변형 ｒｎａ를 포함하는 ｒｎａ 제제
NO2510096T3 (OSRAM) 2009-12-10 2015-03-21
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
WO2011094738A1 (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
CA2799095A1 (en) 2010-05-12 2011-11-17 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
CN103025344B (zh) 2010-05-17 2016-06-29 桑格摩生物科学股份有限公司 新型dna-结合蛋白及其用途
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
US9279103B2 (en) 2010-08-05 2016-03-08 Wisconsin Alumni Research Foundation Simplified basic media for human pluripotent cell culture
US8822663B2 (en) 2010-08-06 2014-09-02 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
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
CA2821992A1 (en) 2010-10-01 2012-04-05 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use 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
US9267123B2 (en) 2011-01-05 2016-02-23 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
WO2012131090A1 (en) 2011-03-31 2012-10-04 Galderma Research & Development Method for treatment of xeroderma pigmentosum
DE12722942T1 (de) 2011-03-31 2021-09-30 Modernatx, Inc. Freisetzung und formulierung von manipulierten nukleinsäuren
US10086043B2 (en) 2011-04-03 2018-10-02 The General Hospital Corporation Efficient protein expression in vivo using modified RNA (MOD-RNA)
KR101982360B1 (ko) 2011-04-05 2019-05-24 셀렉티스 콤팩트 tale-뉴클레아제의 발생 방법 및 이의 용도
JP6158170B2 (ja) * 2011-04-27 2017-07-12 アミリス， インコーポレイテッド ゲノム修飾のための方法
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
US20130040302A1 (en) 2011-07-11 2013-02-14 Thomas J. Burke Methods for cell reprogramming and genome engineering
AU2012289916B2 (en) 2011-08-03 2017-08-03 Phoenix Tissue Repair, Inc. Collagen 7 and related methods
EP3498833B1 (en) * 2011-09-21 2023-08-16 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression
HRP20220250T1 (hr) 2011-10-03 2022-04-29 Modernatx, Inc. Modificirani nukleozidi, nukleotidi i nukleinske kiseline, te njihove uporabe
CA2872033A1 (en) 2011-10-11 2013-04-18 Novartis Ag Recombinant self-replicating polycistronic rna molecules
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
WO2013078199A2 (en) 2011-11-23 2013-05-30 Children's Medical Center Corporation Methods for enhanced in vivo delivery of synthetic, modified rnas
MX354995B (es) 2011-12-05 2018-03-27 Factor Bioscience Inc Metodos y productos para transfeccion de celulas.
US8497124B2 (en) 2011-12-05 2013-07-30 Factor Bioscience Inc. Methods and products for reprogramming cells to a less differentiated state
EP2791159A4 (en) 2011-12-14 2015-10-14 Moderna Therapeutics Inc MODIFIED NUCLEIC ACIDS AND ACUTE TREATMENT USES THEREOF
SI2791160T1 (sl) 2011-12-16 2022-07-29 Modernatx, Inc. Sestave modificirane MRNA
RU2014129863A (ru) 2011-12-21 2016-02-10 Модерна Терапьютикс, Инк. Способы повышения жизнеспособности или увеличения продолжительности жизни органа или экспланта органа
AU2012362113B2 (en) 2011-12-30 2017-08-03 Cellscript, Llc Making and using in vitro-synthesized ssRNA for introducing into mammalian cells to induce a biological or biochemical effect
WO2013142514A1 (en) * 2012-03-19 2013-09-26 Isis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
AU2013243952A1 (en) * 2012-04-02 2014-10-30 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US20130274129A1 (en) 2012-04-04 2013-10-17 Geneart Ag 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
AU2013251649B2 (en) 2012-04-24 2019-04-04 Vcell Therapeutics, Inc. Generating pluripotent cells de novo
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
CN104520424B (zh) 2012-05-13 2019-06-28 美国绿阳生物技术及医药公司 使用合成的信使rna无饲养细胞地衍生人类诱导性多能干细胞
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
KR102315098B1 (ko) * 2012-11-01 2021-10-21 팩터 바이오사이언스 인크. 세포에서 단백질을 발현시키는 방법들과 생성물들
DK2959005T3 (da) 2013-02-22 2022-01-03 Univ Leland Stanford Junior Medicinsk anvendelse relateret til telomer forlængelse
ES2649180T3 (es) * 2013-02-22 2018-01-10 Curevac Ag Combinación de vacunación e inhibición de la ruta PD-1
EP2958992A1 (en) 2013-02-22 2015-12-30 Cellular Dynamics International, Inc. Hepatocyte production via forward programming by combined genetic and chemical engineering
US9393257B2 (en) 2013-03-01 2016-07-19 Regents Of The University Of Minnesota TALEN-based gene correction
DK3011031T3 (da) * 2013-06-17 2020-12-21 Broad Inst Inc Fremføring og anvendelse af crispr-cas-systemerne, vektorer og sammensætninger til levermålretning og -terapi
ES2905257T3 (es) 2013-07-03 2022-04-07 Dicerna Pharmaceuticals Inc Métodos y composiciones para la inhibición específica de alfa-1 antitripsina mediante ARN bicatenario
WO2015038075A1 (en) 2013-09-16 2015-03-19 Agency For Science, Technology And Research Method
WO2015089487A1 (en) 2013-12-12 2015-06-18 Life Technologies Corporation Membrane-penetrating peptides to enhance transfection and compositions and methods for using same
US9840699B2 (en) * 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
EP2918275B1 (en) 2013-12-13 2016-05-18 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
RU2714404C2 (ru) 2014-01-31 2020-02-14 Фэктор Байосайенс Инк. Способы и продукты для получения и доставки нуклеиновых кислот
EP3105317B1 (en) * 2014-02-14 2018-09-19 Cellectis Cells for immunotherapy engineered for targeting antigen present both on immune cells and pathological cells
EP3800248A3 (en) * 2014-04-18 2021-08-04 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
CA2972597A1 (en) * 2014-12-29 2016-07-07 Novartis Ag Methods of making chimeric antigen receptor-expressing cells
CA2976376A1 (en) 2015-02-13 2016-08-18 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
WO2017093804A2 (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
US11512311B2 (en) * 2016-03-25 2022-11-29 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency
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 (OSRAM)
JP7698828B2 (ja) Ｒｎａを編集する方法および組成物
JP7252328B2 (ja) Ｒｎａを編集する方法および組成物
JP2017510542A5 (OSRAM)
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
Lu et al. Multifunctional DNA origami nanoplatforms for drug delivery
Lau et al. CRISPR-based strategies for targeted transgene knock-in and gene correction
JP2022546592A (ja) ゲノム組込みのための方法および組成物
JP2016116520A5 (OSRAM)
FI3599251T3 (fi) Cd20-antigeeniin kohdentuvan kimeerisen antigeenireseptorin rakentaminen ja sillä muunnettujen t-solujen aktiivisuuden tunnistaminen
JP2017537627A5 (OSRAM)
JP2017535258A5 (OSRAM)
JP2017523777A5 (OSRAM)
US20220033792A1 (en) Methods and compositions for cellular reprogramming
JP2012050449A5 (OSRAM)
Liu et al. Viral nanoparticle-encapsidated enzyme and restructured DNA for cell delivery and gene expression
JP2015507625A5 (OSRAM)
Perera et al. Peptide nucleic acids and their role in gene regulation and editing
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
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
Aslesh et al. Restoration of dystrophin expression and correction of Duchenne muscular dystrophy by genome editing