JP2019528284A5 - - Google Patents

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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
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Japan
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composition
uridine
utr
sequence
synthetic
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JP2019509510A
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JP2019528284A (ja
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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
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JP2019509510A 2016-08-17 2017-08-17 核酸産物およびその投与方法 Pending JP2019528284A (ja)

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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)

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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

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JP2019528284A JP2019528284A (ja) 2019-10-10
JP2019528284A5 true JP2019528284A5 (enExample) 2020-09-24

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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

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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)

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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 (59)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3097926T (pt) * 2007-11-01 2020-01-08 Univ Guelph Composições e métodos de potenciação das respostas imunitárias frente a eimeria
KR102121086B1 (ko) 2012-11-01 2020-06-09 팩터 바이오사이언스 인크. 세포에서 단백질을 발현시키는 방법들과 생성물들
US12590321B2 (en) 2016-03-19 2026-03-31 Exuma Biotech Corp. Methods and compositions for genetically modifying and expanding lymphocytes and regulating the activity thereof
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
IL308824A (en) * 2016-08-17 2024-01-01 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
US10676516B2 (en) 2017-05-24 2020-06-09 Pandion Therapeutics, Inc. Targeted immunotolerance
BR112019025224A2 (pt) 2017-05-31 2020-12-08 Ultragenyx Pharmaceutical Inc. Terapêutica para doença de armazenamento de glicogênio tipo iii
CA3079428A1 (en) 2017-11-22 2019-05-31 Modernatx, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
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
US10174092B1 (en) 2017-12-06 2019-01-08 Pandion Therapeutics, Inc. IL-2 muteins
WO2019191341A1 (en) * 2018-03-27 2019-10-03 Factor Bioscience Inc. Nucleic acid-based therapeutics
CN119570787A (zh) * 2018-05-11 2025-03-07 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
AU2019394996B2 (en) 2018-12-06 2025-11-06 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
BR112021023345A2 (pt) 2019-05-20 2022-02-01 Pandion Operations Inc Imunotolerância com alvo em madcam
WO2020252455A1 (en) 2019-06-13 2020-12-17 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
CN110250109B (zh) * 2019-07-01 2021-09-24 上海交通大学医学院附属新华医院 乙醛酸代谢异常相关疾病模型的构建方法、组合物及试剂盒和应用
CA3145425A1 (en) 2019-07-03 2021-01-07 Factor Bioscience Inc. Cationic lipids and uses thereof
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
MX2022001664A (es) 2019-08-09 2022-07-19 Nutcracker Therapeutics Inc Aparato microfluídico y metodos de uso del mismo.
US20220340927A1 (en) * 2019-09-01 2022-10-27 Exuma Biotech Corp. Methods and compositions for the modification and delivery of lymphocytes
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
EP4107187A4 (en) 2020-02-21 2024-07-03 Pandion Operations, Inc. TISSUE-TARGETED IMMUNOTOLERANCE WITH A CD39 EFFECTOR
WO2021173712A1 (en) * 2020-02-24 2021-09-02 The Board Of Regents Of The University Of Texas System Molecular biomarkers and targets for fuchs' endothelial corneal dystrophy and glaucoma
EP4110355A1 (en) * 2020-02-24 2023-01-04 The United States of America, as represented by the Secretary, Department of Health and Human Services Nk cells or t cells expressing hematopoietic growth factor receptors and use for treating cancer
WO2021178448A1 (en) * 2020-03-02 2021-09-10 Motor Life Sciences, Llc Compositions and methods for diagnosing, preventing, and treating amyotrophic lateral sclerosis in patients with hypofunctional anti-trypsin activity
US12351834B2 (en) 2020-03-03 2025-07-08 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
WO2021231549A2 (en) * 2020-05-12 2021-11-18 Factor Bioscience Inc. Engineered gene-editing proteins
CN111714619B (zh) * 2020-07-15 2023-03-14 上海市浦东新区人民医院 成纤维生长因子6在制备缓解非酒精性脂肪性肝炎肝损伤的药物中的应用
WO2022020332A1 (en) 2020-07-21 2022-01-27 Chembeau LLC Diester cosmetic formulations and uses thereof
CA3189601A1 (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
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
BR112023022486A2 (pt) * 2021-05-03 2024-01-16 Ageronix SA Alfa-1-antitripsina (aat) no tratamento e/ou prevenção de distúrbios neurológicos
EP4334330A1 (en) 2021-05-07 2024-03-13 Helix Nanotechnologies, Inc. Modified ribonucleic acids and uses thereof
MX2024002988A (es) * 2021-09-08 2024-06-11 Flagship Pioneering Innovations Vi Llc Composiciones y metodos moduladores de la serpina.
EP4466005A1 (en) * 2022-01-21 2024-11-27 Institut Pasteur De Montevideo Active immunization for reducing osteoarthritic, neuropathic, and cancer pain
CN120303407A (zh) 2022-05-17 2025-07-11 恩维洛普治疗有限责任公司 用于有效体内递送的组合物和方法
WO2024121160A1 (en) * 2022-12-05 2024-06-13 Ethris Gmbh Regulator(s) of energy homeostasis-encoding rna molecule(s) with increased translation efficiency
WO2024229116A2 (en) * 2023-05-01 2024-11-07 Factor Bioscience Inc. Methods for reprogramming and gene editing cells
CN119055669A (zh) * 2023-05-23 2024-12-03 源生生物科技(青岛)有限责任公司 包含miRNA的活性成分及其应用
CN117248020B (zh) * 2023-09-28 2025-06-27 山东大学 Hmgcl作为胶质瘤诊断/预后标志物和治疗靶点的应用
WO2025155772A1 (en) * 2024-01-19 2025-07-24 Gene Company (Pty)Ltd. Alpha-1 anti-trypsin upregulating polynucleotides and method of use and treatment thereof
CN118716283B (zh) * 2024-07-29 2025-01-28 安徽省农业科学院水产研究所 一种保持遗传多样性的鱼类育种方法
CN121476595B (zh) * 2026-01-07 2026-04-10 北京大学人民医院 抗pccb抗体在制备干燥综合征诊断产品中的应用

Family Cites Families (186)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US527565A (en) * 1894-10-16 tripp
US3034507A (en) 1960-05-10 1962-05-15 American Cyanamid Co Intracutaneous injection device
US3539465A (en) 1968-10-08 1970-11-10 Ncr Co Encapsulation of hydrophilic liquid-in-oil emulsions
US3675766A (en) 1970-02-04 1972-07-11 Sol Roy Rosenthal Multiple puncture injector device
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
IE61148B1 (en) * 1988-03-10 1994-10-05 Ici Plc Method of detecting nucleotide sequences
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
US5849686A (en) 1991-03-11 1998-12-15 Creative Biomolecules, Inc. Morphogen-induced liver regeneration
US5656593A (en) 1991-03-11 1997-08-12 Creative Biomolecules, Inc. Morphogen induced periodontal tissue regeneration
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
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
EP0894004B2 (en) 1996-03-22 2007-02-21 Curis, Inc. Method for enhancing functional recovery of motor coordination, speech or sensory perception after central nervous system ischemia or 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
US6413544B1 (en) 1996-08-19 2002-07-02 The United States Of America As Represented By The Department Of Health And Human Services Liposome complexes for increased systemic delivery
TW520297B (en) 1996-10-11 2003-02-11 Sequus Pharm Inc Fusogenic liposome composition and method
US6653104B2 (en) 1996-10-17 2003-11-25 Immunomedics, Inc. Immunotoxins, comprising an internalizing antibody, directed against malignant and normal cells
CA2277278A1 (en) 1997-01-10 1998-07-16 Life Technologies, Inc. Embryonic stem cell serum replacement
US6316260B1 (en) 1997-08-22 2001-11-13 Bernina Biosystems Gmbh Tetraether lipid derivatives and liposomes and lipid agglomerates containing tetraether lipid derivatives, and use thereof
US20030083272A1 (en) 1997-09-19 2003-05-01 Lahive & Cockfield, Llp Sense mrna therapy
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
CA2376128C (en) 1999-06-04 2009-01-06 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
AU2001294750C1 (en) 2000-09-26 2008-09-18 Idera Pharmaceuticals, 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
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
JP4579911B2 (ja) 2003-06-03 2010-11-10 アイシス・ファーマシューティカルズ・インコーポレイテッド スルビビン発現の調節
US20070134796A1 (en) 2005-07-26 2007-06-14 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
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
ATE549395T1 (de) 2004-09-08 2012-03-15 Wisconsin Alumni Res Found Medium und kultur von embryonalen stammzellen
AU2005282414C1 (en) 2004-09-08 2011-04-07 Wisconsin Alumni Research Foundation Culturing human 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
BRPI0606934A2 (pt) 2005-01-25 2017-07-11 Cell Therapeutics Inc Conjugado de proteína biologicamente ativa, composição, molécula de dna quimérica, vetor, célula, e, métodos para preparar conjugado de proteína biologicamente ativa, e para determinar se um dado conjugado de proteína exibe uma meia-vida de plasma modificada comparada com a meia-vida intrínseca do polipeptídeo biologicamente ativo não conjungado
WO2007006808A1 (en) 2005-07-13 2007-01-18 Novo Nordisk Health Care Ag Host cell protein knock-out cells for production of therapeutic proteins
US8323666B2 (en) 2005-08-01 2012-12-04 Allergan, Inc. Botulinum toxin compositions
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
EP1962719A4 (en) 2005-08-29 2011-05-04 Technion Res And Dev Of Foundation Ltd MEDIA FOR BREEDING 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
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
WO2008073856A2 (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
WO2008086529A2 (en) 2007-01-11 2008-07-17 Yale University Compositions and methods for targeted inactivation of hiv cell surface receptors
WO2008097926A2 (en) 2007-02-02 2008-08-14 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
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
CN105861443A (zh) 2007-04-07 2016-08-17 怀特黑德生物医学研究所 体细胞重编程
WO2008151845A2 (en) * 2007-06-15 2008-12-18 Charite - Universitätsmedizin Berlin Methods for producing glycosylated human alpha-1 antitrypsin (a1at) in mammalian host cells
JP2008307007A (ja) 2007-06-15 2008-12-25 Bayer Schering Pharma Ag 出生後のヒト組織由来未分化幹細胞から誘導したヒト多能性幹細胞
JP5558097B2 (ja) 2007-12-10 2014-07-23 国立大学法人京都大学 効率的な核初期化方法
WO2009077509A1 (en) 2007-12-14 2009-06-25 Dsm Ip Assets B.V. Sol-gel process with a protected catalyst
EP2072618A1 (en) 2007-12-14 2009-06-24 Johannes Gutenberg-Universität Mainz Use of RNA for reprogramming somatic cells
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
MX2010010165A (es) 2008-03-17 2010-11-25 Scripps Research Inst Procedimientos quimicos y geneticos combinados para generacion de celulas madre pluripotentes inducidas.
US20110045001A1 (en) 2008-03-28 2011-02-24 Biontex Laboratories Gmbh Transfection results of non-viral gene delivery systems by influencing of the innate immune system
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
JP2011160661A (ja) 2008-06-02 2011-08-25 Kyowa Hakko Kirin Co Ltd 血球細胞の初期化法
EP3279314A1 (en) 2008-06-04 2018-02-07 Cellular Dynamics International, Inc. Methods for the production of ips cells using non-viral approach
GB2460552B (en) 2008-06-05 2011-09-07 Iti Scotland Ltd Stem cell culture media and methods
US8669048B2 (en) 2008-06-24 2014-03-11 Parkinson's Institute Pluripotent cell lines and methods of use thereof
US20100184033A1 (en) 2008-07-16 2010-07-22 West Michael D Methods to accelerate the isolation of novel cell strains from pluripotent stem cells and cells obtained thereby
EP2151248A1 (en) 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
WO2010022194A2 (en) 2008-08-20 2010-02-25 Virxsys Corporation Compositions and methods for generation of pluripotent stem cells
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
DK2356221T3 (en) 2008-10-24 2019-02-18 Wisconsin Alumni Res Found Pluripotent stem cells obtained by non-viral reprogramming
BRPI0916150B1 (pt) 2008-11-18 2019-09-24 3M Innovative Properties Company Arranjo de microagulhas ocas
EP2881461A1 (en) 2008-11-21 2015-06-10 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
EP2421957B1 (en) 2009-04-22 2020-11-18 Viacyte, Inc. Cell compositions derived from dedifferentiated reprogrammed cells
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
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
CA2769670C (en) 2009-07-31 2018-10-02 Ethris Gmbh Rna with a combination of unmodified and modified nucleotides for protein expression
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
WO2011056971A2 (en) 2009-11-04 2011-05-12 Cellular Dynamics International, Inc. Episomal reprogramming with chemicals
AU2010319555A1 (en) 2009-11-11 2012-06-14 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
US8808982B2 (en) 2009-12-07 2014-08-19 Cellscript, Llc Compositions and methods for reprogramming eukaryotic cells
WO2011072246A2 (en) 2009-12-10 2011-06-16 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
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
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
CA2799095A1 (en) 2010-05-12 2011-11-17 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
US8048675B1 (en) 2010-05-12 2011-11-01 Ipierian, Inc. Integration-free human induced pluripotent stem cells from blood
EP2392208B1 (en) 2010-06-07 2016-05-04 Helmholtz Zentrum München Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Fusion proteins comprising a DNA-binding domain of a Tal effector protein and a non-specific cleavage domain of a restriction nuclease and their use
AU2011265733B2 (en) 2010-06-14 2014-04-17 Iowa State University Research Foundation, Inc. Nuclease activity of TAL effector and Foki fusion protein
US9279103B2 (en) 2010-08-05 2016-03-08 Wisconsin Alumni Research Foundation Simplified basic media for human pluripotent cell culture
WO2012019168A2 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
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
EP2625189B1 (en) 2010-10-01 2018-06-27 ModernaTX, 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
JP5888753B2 (ja) 2010-11-04 2016-03-22 国立大学法人京都大学 効率的な人工多能性幹細胞の樹立方法
WO2012094132A1 (en) 2011-01-05 2012-07-12 Sangamo Biosciences, Inc. Methods and compositions for gene correction
CN103502436A (zh) 2011-03-07 2014-01-08 麻省理工学院 用核酸转染细胞的方法
AU2012236099A1 (en) 2011-03-31 2013-10-03 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
WO2012131090A1 (en) 2011-03-31 2012-10-04 Galderma Research & Development Method for treatment of xeroderma pigmentosum
EP3460064B8 (en) 2011-04-03 2024-03-20 The General Hospital Corporation d/b/a Massachusetts General Hospital Efficient protein expression in vivo using modified rna (mod-rna)
CA3111953C (en) 2011-04-05 2023-10-24 Cellectis Method for the generation of compact tale-nucleases and uses thereof
AU2012249390B2 (en) * 2011-04-27 2017-03-30 Amyris, Inc. Methods for genomic modification
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
JP2014520551A (ja) 2011-07-11 2014-08-25 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 細胞のリプログラミング方法およびゲノムの改変方法
EP2753704A4 (en) 2011-08-03 2015-07-08 Lotus Tissue Repair Inc COLLAGEN 7 AND ASSOCIATED METHODS
CA2848417C (en) * 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
DE19216461T1 (de) 2011-10-03 2021-10-07 Modernatx, Inc. Modifizierte nukleoside, nukleotide und nukleinsäuren und verwendungen davon
RU2014118727A (ru) 2011-10-11 2015-11-20 Новартис Аг Рекомбинантные самореплицирующиеся полицистронные молекулы рнк
ES2670495T3 (es) 2011-10-11 2018-05-30 Inserm - Institut National De La Santé Et De La Recherche Médicale Tratamiento de salto de exones para epidermólisis ampollosa distrófica
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
AU2012347919B2 (en) 2011-12-05 2017-02-02 Factor Bioscience Inc. Methods and products for transfecting cells
WO2013090186A1 (en) 2011-12-14 2013-06-20 modeRNA Therapeutics Modified nucleic acids, and acute care uses thereof
CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
CN104968354A (zh) 2011-12-21 2015-10-07 现代治疗公司 增加器官或器官外植体的活力或寿命的方法
EP3144389B1 (en) 2011-12-30 2018-05-09 Cellscript, Llc Making and using in vitro-synthesized ssrna for introducing into mammalian cells to induce a biological or biochemical effect
US9340784B2 (en) * 2012-03-19 2016-05-17 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
WO2013152220A2 (en) 2012-04-04 2013-10-10 Life Technologies Corporation Tal-effector assembly platform, customized services, kits and assays
US20150211004A1 (en) 2012-04-20 2015-07-30 Agency For Science, Technology And Research Rnai-based therapies for cardiomyopathies, muscular dystrophies and laminopathies
SG11201407768QA (en) 2012-04-24 2015-01-29 Brigham & Womens Hospital Generating pluripotent cells de novo
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
EP3561054B1 (en) 2012-05-13 2023-07-05 Allele Biotechnology And Pharmaceuticals, Inc. Feeder-free derivation of human-induced pluripotent stem cells with synthetic messenger rna
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
US20140031295A1 (en) 2012-07-19 2014-01-30 Lotus Tissue Repair, Inc. Recombinant C7 and Methods of Use
AU2013329186B2 (en) * 2012-10-10 2019-02-14 Sangamo Therapeutics, Inc. T cell modifying compounds and uses thereof
KR102121086B1 (ko) 2012-11-01 2020-06-09 팩터 바이오사이언스 인크. 세포에서 단백질을 발현시키는 방법들과 생성물들
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
US9974845B2 (en) 2013-02-22 2018-05-22 Curevac Ag Combination of vaccination and inhibition of the PD-1 pathway
AU2014218667A1 (en) 2013-02-22 2015-10-08 The Board Of Trustees Of The Leland Stanford Junior University Compounds, compositions, methods, and kits relating to telomere extension
WO2014134412A1 (en) 2013-03-01 2014-09-04 Regents Of The University Of Minnesota Talen-based gene correction
RU2716420C2 (ru) * 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени
US9458457B2 (en) 2013-07-03 2016-10-04 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
US20150165054A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
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
EP3053585A1 (en) 2013-12-13 2016-08-10 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
EP3099801B1 (en) * 2014-01-31 2020-03-18 Factor Bioscience Inc. Synthetic rna for use in the treatment of dystrophic epidermolysis bullosa
WO2015121454A1 (en) * 2014-02-14 2015-08-20 Cellectis Cells for immunotherapy engineered for targeting antigen present both on immune cells and pathological cells
WO2015161276A2 (en) * 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
PL4023249T3 (pl) * 2014-04-23 2025-03-10 Modernatx, Inc. Szczepionki z kwasem nukleinowym
US20170335331A1 (en) * 2014-10-31 2017-11-23 The Trustees Of The University Of Pennsylvania Altering Gene Expression in CART Cells and Uses Thereof
RU2021118125A (ru) * 2014-12-29 2022-04-06 Новартис Аг Способы получения экспрессирующих химерный антигенный рецептор клеток
CA2976376A1 (en) 2015-02-13 2016-08-18 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
CN109715801B (zh) * 2015-12-01 2022-11-01 克里斯普治疗股份公司 用于治疗α1抗胰蛋白酶缺乏的材料和方法
EP3423580A1 (en) * 2016-03-04 2019-01-09 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
EP4628587A3 (en) * 2016-04-15 2026-02-25 Memorial Sloan Kettering Cancer Center Transgenic t cell and chimeric antigen receptor t cell compositions and related methods
IL308824A (en) 2016-08-17 2024-01-01 Factor Bioscience Inc Nucleic acid products and methods of administration thereof

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