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 (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) CN109803977B (enExample)
AU (2) AU2017312113B2 (enExample)
CA (1) CA3033788A1 (enExample)
IL (2) IL308824A (enExample)
WO (1) WO2018035377A1 (enExample)

Families Citing this family (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ601880A (en) * 2007-11-01 2013-12-20 Univ Arkansas Compositions and methods of enhancing immune responses to eimeria
MX363017B (es) * 2012-11-01 2019-03-04 Factor Bioscience Inc Métodos y productos para la expresión de proteínas en células.
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
IL308824A (en) * 2016-08-17 2024-01-01 Factor Bioscience Inc Nucleic acid products and methods of administration thereof
AU2018234810B2 (en) 2017-03-15 2023-05-11 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
US10676516B2 (en) 2017-05-24 2020-06-09 Pandion Therapeutics, Inc. Targeted immunotolerance
EP3630964B1 (en) 2017-05-31 2025-08-13 Ultragenyx Pharmaceutical Inc. Therapeutics for glycogen storage disease type iii
US11859215B2 (en) 2017-11-22 2024-01-02 Modernatx, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
US10174092B1 (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
WO2019191341A1 (en) * 2018-03-27 2019-10-03 Factor Bioscience Inc. Nucleic acid-based therapeutics
WO2019217941A1 (en) * 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of suppressing 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
JP7558167B2 (ja) 2018-12-03 2024-09-30 ボード オブ リージェンツ,ザ ユニバーシティ オブ テキサス システム オリゴベンズアミド類似体およびがん処置におけるそれらの使用
ES2960692T3 (es) 2018-12-06 2024-03-06 Arcturus Therapeutics Inc Composiciones y métodos para el tratamiento de la deficiencia de ornitina transcarbamilasa
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
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
CN116057181A (zh) * 2019-09-01 2023-05-02 埃克苏马生物技术公司 用于修饰和递送淋巴细胞的方法和组合物
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
KR102490457B1 (ko) * 2019-11-26 2023-01-19 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 BIOMARKERS AND MOLECULAR TARGETS FOR FUCHS CORNEAL ENDOTHELIAL 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
US12351834B2 (en) 2020-03-03 2025-07-08 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
EP4185265A4 (en) 2020-07-21 2024-06-26 Chembeau LLC DIESTER COSMETIC FORMULATIONS AND USES THEREOF
MX2023001028A (es) 2020-07-24 2023-04-24 Massachusetts Gen Hospital Particulas similares a virus potenciadas y metodos de uso de las mismas para suministro a celulas.
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
CN117412986A (zh) * 2021-04-02 2024-01-16 克里斯托生物技术股份有限公司 用于癌症疗法的病毒载体
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
BR112023022486A2 (pt) * 2021-05-03 2024-01-16 Ageronix SA Alfa-1-antitripsina (aat) no tratamento e/ou prevenção de distúrbios neurológicos
IL308298A (en) 2021-05-07 2024-01-01 Helix Nanotechnologies Inc Modified ribonucleic acids and their uses
WO2023139542A1 (en) * 2022-01-21 2023-07-27 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
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
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
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
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
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
WO1998030679A1 (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
CA2304982A1 (en) 1997-09-19 1999-03-25 Sequitur, Inc. Sense mrna therapy
NZ512244A (en) 1998-11-12 2003-12-19 Invitrogen Corp Polycationic transfection reagents for introducing anions into a cell
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
AU5461300A (en) 1999-06-04 2000-12-28 Georgia Tech Research Corporation Devices and methods 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
ES2298269T3 (es) 2000-09-26 2008-05-16 Idera Pharmaceuticals, Inc. Modulacion de la actividad inmunoestimulante de analogos oligonucleotidicos inmunoestimulantes mediante cambios quimicos posicionales.
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
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
US20030138951A1 (en) 2001-10-18 2003-07-24 Li Yin 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
EP1633307A4 (en) 2003-06-03 2009-06-24 Isis Pharmaceuticals Inc Modulation of survivin expression
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
AU2005282510B2 (en) 2004-09-08 2010-12-02 Wisconsin Alumni Research Foundation Medium and culture of embryonic stem cells
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
RU2007132188A (ru) 2005-01-25 2009-03-10 Селл Терапьютикс, Инк. (Us) Конъюгаты биологически активных белков, имеющие модифицированное время полужизни in vivo
WO2007006808A1 (en) 2005-07-13 2007-01-18 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
TR201909609T4 (tr) 2005-08-23 2019-07-22 Univ Pennsylvania Modifiye edilmiş nükleosidleri içeren rna ve kullanım yöntemleri.
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
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
EA018039B1 (ru) 2005-12-13 2013-05-30 Киото Юниверсити Ядерный фактор перепрограммирования
US8278104B2 (en) 2005-12-13 2012-10-02 Kyoto University Induced pluripotent stem cells produced with Oct3/4, Klf4 and Sox2
US8129187B2 (en) 2005-12-13 2012-03-06 Kyoto University Somatic cell reprogramming by retroviral vectors encoding Oct3/4. Klf4, c-Myc and Sox2
KR20080078010A (ko) 2005-12-22 2008-08-26 체에스엘 베링 게엠베하 옥타노에이트-감소된 사람 알부민
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
JP5813321B2 (ja) 2007-03-23 2015-11-17 ウィスコンシン アラムニ リサーチ ファンデーション 体細胞の再プログラム化
MX348010B (es) 2007-04-07 2017-05-23 Whitehead Inst Biomedical Res Reprogramacion de celulas somaticas.
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
KR101532442B1 (ko) 2007-12-10 2015-06-29 고쿠리츠 다이가쿠 호진 교토 다이가쿠 효율적인 핵 초기화 방법
EP2072618A1 (en) 2007-12-14 2009-06-24 Johannes Gutenberg-Universität Mainz Use of RNA for reprogramming somatic cells
US20110021335A1 (en) 2007-12-14 2011-01-27 Pascal Jozef Paul Buskens Sol-gel process with a protected catalyst
JP5529754B2 (ja) 2007-12-21 2014-06-25 ストライカー コーポレイション Nogginに対する感受性が低下したBMP変異体
KR101679082B1 (ko) 2008-03-17 2016-11-23 더 스크립스 리서치 인스티튜트 유도 만능 줄기 세포 생성을 위한 화학적 및 유전적 조합 접근법
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
ES2587395T3 (es) 2008-06-04 2016-10-24 Cellular Dynamics International, Inc. Procedimientos para la producción de células IPS usando un enfoque no vírico
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
CN102239249A (zh) 2008-10-24 2011-11-09 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
WO2010059605A2 (en) 2008-11-18 2010-05-27 3M Innovative Properties Company Hollow microneedle array and method
EP2192174B1 (en) 2008-11-21 2015-11-11 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Reprogramming cells toward a pluripotent state
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
EP2412800A1 (en) 2010-07-29 2012-02-01 Koninklijke Nederlandse Akademie van Wetenschappen Liver organoid, uses thereof and culture method for obtaining them
US20100209404A1 (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
ES2849181T3 (es) 2009-04-22 2021-08-16 Viacyte Inc Composiciones celulares derivadas de células reprogramadas desdiferenciadas
WO2010123501A1 (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
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
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"
JP5898086B2 (ja) 2009-11-04 2016-04-06 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 化学物質を用いるエピソームリプログラミング
US20110189137A1 (en) 2009-11-11 2011-08-04 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
CN107090436B (zh) 2009-12-07 2021-11-30 宾夕法尼亚州大学信托人 用于重编程细胞的包含纯化的经修饰的rna的rna制剂
AU2010327998B2 (en) 2009-12-10 2015-11-12 Iowa State University Research Foundation, Inc. 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
US20130071365A1 (en) 2010-02-16 2013-03-21 Kyushu University, National University Corporation Induced hepatocytes
WO2011110886A1 (en) 2010-03-09 2011-09-15 Biolamina Ab Composition and method for enabling proliferation of pluripotent human stem cells
US8802438B2 (en) 2010-04-16 2014-08-12 Children's Medical Center Corporation Compositions, kits, and methods for making induced pluripotent stem cells using synthetic modified RNAs
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
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins 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
JP2013534417A (ja) 2010-06-14 2013-09-05 アイオワ ステート ユニバーシティ リサーチ ファウンデーション,インコーポレーティッド Talエフェクターとfokiの融合タンパク質のヌクレアーゼ活性
AU2011285531B2 (en) 2010-08-05 2015-04-30 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
EP2616540A4 (en) 2010-09-14 2014-02-19 Univ Kyoto METHOD FOR EFFICIENT MANUFACTURE OF INDUCED PLURIPOTENTER STEM CELLS
PL3590949T3 (pl) 2010-10-01 2022-08-29 Modernatx, Inc. Kwasy rybonukleinowe zawierające n1-metylo-pseudouracyle i ich zastosowania
EP2625278A1 (en) 2010-10-08 2013-08-14 Regents of the University of Minnesota A method to increase gene targeting frequency
WO2012060473A1 (en) 2010-11-04 2012-05-10 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
AU2012225497A1 (en) 2011-03-07 2013-10-24 Massachusetts Institute Of Technology Methods for transfecting cells with nucleic acids
DE12722942T1 (de) 2011-03-31 2021-09-30 Modernatx, Inc. Freisetzung und formulierung von manipulierten nukleinsäuren
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)
CA2832534C (en) 2011-04-05 2022-01-04 Julien Valton Method for the generation of compact tale-nucleases and uses thereof
BR112013025567B1 (pt) * 2011-04-27 2021-09-21 Amyris, Inc Métodos para modificação genômica
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
WO2013003475A1 (en) 2011-06-27 2013-01-03 Cellscript, Inc. Inhibition of innate immune response
US20130040302A1 (en) 2011-07-11 2013-02-14 Thomas J. Burke Methods for cell reprogramming and genome engineering
EP2753704A4 (en) 2011-08-03 2015-07-08 Lotus Tissue Repair Inc COLLAGEN 7 AND ASSOCIATED METHODS
JP6185916B2 (ja) * 2011-09-21 2017-08-23 サンガモ セラピューティクス, インコーポレイテッド 導入遺伝子発現を制御するための方法および組成物
RS62993B1 (sr) 2011-10-03 2022-03-31 Modernatx Inc Modifikovani nukleozidi, nukleotidi, i nukleinske kiseline, i njihove upotrebe
AU2012322704B2 (en) 2011-10-11 2017-09-07 Novartis Ag Recombinant self-replicating polycistronic RNA molecules
EP2766480B1 (en) 2011-10-11 2018-03-14 INSERM - Institut National de la Santé et de la Recherche Médicale 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
KR20250035015A (ko) 2011-12-05 2025-03-11 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
EP2791159A4 (en) 2011-12-14 2015-10-14 Moderna Therapeutics Inc MODIFIED NUCLEIC ACIDS AND ACUTE TREATMENT USES THEREOF
PT2791160T (pt) 2011-12-16 2022-07-04 Modernatx Inc Composições arnm modificado
CA2859691A1 (en) 2011-12-21 2013-06-27 Moderna Therapeutics, Inc. Methods of increasing the viability or longevity of an organ or organ explant
LT3421601T (lt) 2011-12-30 2020-01-10 Cellscript, Llc In vitro susintetintos ssrnr gamyba ir panaudojimas įvedimui į žinduolių ląsteles biologinio arba biocheminio poveikio sukėlimui
WO2013142514A1 (en) * 2012-03-19 2013-09-26 Isis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
AU2013243955B2 (en) 2012-04-02 2018-02-22 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US20130274129A1 (en) 2012-04-04 2013-10-17 Geneart Ag Tal-effector assembly platform, customized services, kits and assays
US20150211004A1 (en) 2012-04-20 2015-07-30 Agency For Science, Technology And Research Rnai-based therapies for cardiomyopathies, muscular dystrophies and laminopathies
SG10201809901SA (en) 2012-04-24 2018-12-28 Vcell Therapeutics Inc Generating Pluripotent Cells De Novo
KR102081811B1 (ko) 2012-05-13 2020-02-26 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
EP3763810A3 (en) * 2012-10-10 2021-07-14 Sangamo Therapeutics, Inc. T cell modifying compounds and uses thereof
MX363017B (es) 2012-11-01 2019-03-04 Factor Bioscience Inc Métodos y productos para la expresión de proteínas en células.
CN105164269A (zh) 2013-02-22 2015-12-16 里兰斯坦福初级大学理事会 与端粒延伸相关的化合物、组合物、方法和试剂盒
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
SG10201710473VA (en) * 2013-02-22 2018-02-27 Curevac Ag Combination of vaccination and inhibition of the pd-1 pathway
JP6480874B2 (ja) 2013-03-01 2019-03-13 リージェンツ・オブ・ザ・ユニバーシティ・オブ・ミネソタRegents Of The University Of Minnesota Talenに基づく遺伝子修正
EP3011031B1 (en) * 2013-06-17 2020-09-30 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
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
SG11201601939VA (en) 2013-09-16 2016-04-28 Agency Science Tech & Res Method
BR112016013516A8 (pt) 2013-12-12 2020-05-19 Life Technologies Corp complexo de transfecção e seu uso para aumentar transporte de molécula de carga em células e para o tratamento de câncer e terapia gênica, bem como método in vitro para fornecer um poliânion a uma célula
US20150166984A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
US20150167017A1 (en) 2013-12-13 2015-06-18 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
EP4249036A3 (en) 2014-01-31 2023-10-25 Factor Bioscience Inc. Methods and products for nucleic acid production and delivery
EP3105317B1 (en) * 2014-02-14 2018-09-19 Cellectis Cells for immunotherapy engineered for targeting antigen present both on immune cells and pathological cells
AU2015247323B2 (en) * 2014-04-18 2021-07-01 Editas Medicine, Inc. CRISPR-Cas-related methods, compositions and components for cancer immunotherapy
CA3177878A1 (en) * 2014-04-23 2015-10-29 Modernatx, Inc. Nucleic acid vaccines
AU2015374296B2 (en) * 2014-12-29 2021-09-02 Novartis Ag Methods of making chimeric antigen receptor-expressing cells
US11241505B2 (en) 2015-02-13 2022-02-08 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
EP3384024B1 (en) * 2015-12-01 2022-02-02 CRISPR Therapeutics AG Materials and methods for treatment of alpha-1 antitrypsin deficiency
EP3423580A1 (en) * 2016-03-04 2019-01-09 Editas Medicine, Inc. Crispr-cpf1-related methods, compositions and components for cancer immunotherapy
EP3433364A1 (en) * 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
IL308824A (en) * 2016-08-17 2024-01-01 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
Shu et al. Stable RNA nanoparticles as potential new generation drugs for cancer therapy
JP2017510542A5 (enExample)
US11186825B2 (en) Compositions and methods for evaluating and modulating immune responses by detecting and targeting POU2AF1
Xiong et al. CRISPR/Cas9 for human genome engineering and disease research
US20180100201A1 (en) Tumor and microenvironment gene expression, compositions of matter and methods of use thereof
Sambasivan et al. Embryonic founders of adult muscle stem cells are primed by the determination gene Mrf4
Lu et al. Multifunctional DNA origami nanoplatforms for drug delivery
US20190106679A1 (en) Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
JP2017537627A5 (enExample)
JP2017535258A5 (enExample)
JP2009159971A5 (enExample)
JP2012050449A5 (enExample)
WO2017075478A2 (en) Compositions and methods for evaluating and modulating immune responses by use of immune cell gene signatures
Liu et al. Viral nanoparticle-encapsidated enzyme and restructured DNA for cell delivery and gene expression
JP2015513897A5 (enExample)
JP2015507625A5 (enExample)
Perera et al. Peptide nucleic acids and their role in gene regulation and editing
US20180119122A1 (en) Methods and compositions for cellular reprogramming
Wang et al. CRISPR applications in cancer diagnosis and treatment
Meng et al. Optimization of CRISPR–Cas system for clinical cancer therapy
Brooks et al. Challenges of gene editing therapies for genodermatoses
Yan et al. Advances in RNA-based cancer therapeutics: pre-clinical and clinical implications