MX2019002498A - Metodos y productos para la expresion de proteinas en celulas. - Google Patents

Metodos y productos para la expresion de proteinas en celulas.

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Publication number
MX2019002498A
MX2019002498A MX2019002498A MX2019002498A MX2019002498A MX 2019002498 A MX2019002498 A MX 2019002498A MX 2019002498 A MX2019002498 A MX 2019002498A MX 2019002498 A MX2019002498 A MX 2019002498A MX 2019002498 A MX2019002498 A MX 2019002498A
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Mexico
Prior art keywords
methods
cells
products
proteins
nucleic acids
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MX2019002498A
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English (en)
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Matthew Angel
Christopher Rohde
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Factor Bioscience Inc
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Application filed by Factor Bioscience Inc filed Critical Factor Bioscience Inc
Publication of MX2019002498A publication Critical patent/MX2019002498A/es

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Abstract

La presente invención se refiere, en parte, a ácidos nucleicos que codifican proteínas, agentes terapéuticos que comprenden ácidos nucleicos que codifican proteínas, métodos para inducir a células a expresar proteínas utilizando ácidos nucleicos, kits y dispositivos para transfectar, realizar la edición genética y reprogramar células, y células, organismos y agentes terapéuticos producidos usando estos métodos, kits y dispositivos. Se describen métodos y productos para alterar la secuencia de ADN de una célula, así como también métodos y productos para inducir a células a expresar proteínas, mediante el uso de moléculas de ARN sintético. También se describen agentes terapéuticos que comprenden ácidos nucleicos que codifican proteínas de edición de genes.
MX2019002498A 2012-11-01 2015-04-27 Metodos y productos para la expresion de proteinas en celulas. MX2019002498A (es)

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US201261721302P 2012-11-01 2012-11-01
US201361785404P 2013-03-14 2013-03-14
US201361842874P 2013-07-03 2013-07-03

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MX2019002498A MX2019002498A (es) 2012-11-01 2015-04-27 Metodos y productos para la expresion de proteinas en celulas.

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EP (2) EP3786298A1 (es)
JP (4) JP6510416B2 (es)
KR (4) KR102315098B1 (es)
CN (1) CN104769112A (es)
AU (3) AU2013337651B2 (es)
BR (2) BR122019025678B1 (es)
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Families Citing this family (106)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10347710B4 (de) 2003-10-14 2006-03-30 Johannes-Gutenberg-Universität Mainz Rekombinante Impfstoffe und deren Verwendung
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
ES2862955T3 (es) 2010-10-01 2021-10-08 Modernatx Inc Acidos nucleicos manipulados y métodos de uso de los mismos
US8710200B2 (en) 2011-03-31 2014-04-29 Moderna Therapeutics, Inc. Engineered nucleic acids encoding a modified erythropoietin and their expression
DK2714071T3 (da) 2011-05-24 2019-09-16 Biontech Rna Pharmaceuticals Gmbh Individualiserede vacciner mod cancer
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
WO2013052523A1 (en) 2011-10-03 2013-04-11 modeRNA Therapeutics Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
CN104114572A (zh) 2011-12-16 2014-10-22 现代治疗公司 经修饰的核苷、核苷酸和核酸组合物
WO2013143555A1 (en) 2012-03-26 2013-10-03 Biontech Ag Rna formulation for immunotherapy
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
EP2833920A2 (en) 2012-04-02 2015-02-11 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9878056B2 (en) 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
US9539307B2 (en) 2012-09-17 2017-01-10 The Research Institute At Nationwide Children's Hospital Compositions and methods for treating amyotrophic lateral sclerosis
WO2014071219A1 (en) 2012-11-01 2014-05-08 Factor Bioscience Inc. Methods and products for expressing proteins in cells
PL2922554T3 (pl) 2012-11-26 2022-06-20 Modernatx, Inc. Na zmodyfikowany na końcach
EP3417874A1 (en) 2012-11-28 2018-12-26 BioNTech RNA Pharmaceuticals GmbH Individualized vaccines for cancer
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
WO2014180490A1 (en) 2013-05-10 2014-11-13 Biontech Ag Predicting immunogenicity of t cell epitopes
AU2014274840B2 (en) * 2013-06-05 2020-03-12 Duke University RNA-guided gene editing and gene regulation
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
SI3039146T1 (sl) 2013-08-27 2020-10-30 Research Institute At Nationwide Children's Hospital Proizvodi in postopki za zdravljenje amiotrofične lateralne skleroze
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052521A1 (en) 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
US9725719B2 (en) 2013-11-05 2017-08-08 The Research Institute At Nationwide Children's Hospital Compositions and methods for inhibiting NF-κB and SOD-1 to treat amyotrophic lateral sclerosis
KR102523466B1 (ko) 2013-11-07 2023-04-20 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
EP3116533B1 (en) * 2014-03-12 2020-08-12 Precision Biosciences, Inc. Dystrophin gene exon deletion using engineered nucleases
SG11201608798YA (en) 2014-04-23 2016-11-29 Modernatx Inc Nucleic acid vaccines
EP3140403A4 (en) * 2014-05-09 2017-12-20 Université Laval Prevention and treatment of alzheimer's disease by genome editing using the crispr/cas system
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016045732A1 (en) 2014-09-25 2016-03-31 Biontech Rna Pharmaceuticals Gmbh Stable formulations of lipids and liposomes
US10370673B2 (en) 2014-09-26 2019-08-06 Purecircle Sdn Bhd Single nucleotide polymorphism (SNP) markers for stevia
RU2020108189A (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
US10195261B2 (en) 2014-11-26 2019-02-05 VaxLiant, LLC Adjuvant compositions and related methods
ES2961965T3 (es) * 2014-11-26 2024-03-14 Huvepharma Inc Composiciones adyuvantes y procedimientos relacionados
WO2016089433A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
WO2016128060A1 (en) 2015-02-12 2016-08-18 Biontech Ag Predicting t cell epitopes useful for vaccination
AU2016218977C1 (en) 2015-02-13 2023-03-23 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
US9636397B2 (en) 2015-03-24 2017-05-02 VaxLiant, LLC Adjuvant compositions and related methods
CN107787367B (zh) 2015-04-06 2021-10-26 里兰斯坦福初级大学理事会 用于crispr/cas介导的基因调控的化学修饰的引导rna
US10676735B2 (en) 2015-07-22 2020-06-09 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
AU2016299271B2 (en) * 2015-07-25 2022-09-22 Habib FROST A system, device and a method for providing a therapy or a cure for cancer and other pathological states
IL288662B2 (en) 2015-08-25 2023-09-01 Univ Duke Preparations and methods for improving specificity in genomic engineering using RNA-guided endonucleases
WO2017059902A1 (en) 2015-10-07 2017-04-13 Biontech Rna Pharmaceuticals Gmbh 3' utr sequences for stabilization of rna
WO2017066797A1 (en) 2015-10-16 2017-04-20 Modernatx, Inc. Trinucleotide mrna cap analogs
ES2914225T3 (es) 2015-10-16 2022-06-08 Modernatx Inc Análogos de cap de ARNm con enlace de fosfato modificado
IL258821B (en) 2015-10-23 2022-07-01 Harvard College Nucleobase editors and their uses
EP3384038A4 (en) * 2015-11-23 2019-12-04 The Regents of The University of Colorado, A Body Corporate METHODS AND COMPOSITIONS FOR CELL REPROGRAMMING
CN105494263B (zh) * 2015-12-25 2018-11-30 哈尔滨医科大学 一种产生ho-1/app/psen1三转基因阿尔茨海默病小鼠模型的方法
EP3411078A1 (en) * 2016-02-02 2018-12-12 Crispr Therapeutics AG Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
EP3416689B1 (en) * 2016-02-18 2023-01-18 CRISPR Therapeutics AG Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
US11530253B2 (en) 2016-02-25 2022-12-20 The Children's Medical Center Corporation Customized class switch of immunoglobulin genes in lymphoma and hybridoma by CRISPR/CAS9 technology
EP3429632B1 (en) * 2016-03-16 2023-01-04 CRISPR Therapeutics AG Materials and methods for treatment of hereditary haemochromatosis
ES2835051T3 (es) * 2016-03-31 2021-06-21 Ethris Gmbh Secuencias de UTR mínimas novedosas
US20200172935A1 (en) * 2016-04-16 2020-06-04 Ohio State Innovation Foundation Modified cpf1 mrna, modified guide rna, and uses thereof
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
JP7033583B2 (ja) * 2016-07-13 2022-03-10 バーテックス ファーマシューティカルズ インコーポレイテッド ゲノム編集効率を高めるための方法、組成物及びキット
IL264565B1 (en) 2016-08-03 2024-03-01 Harvard College Adenosine nuclear base editors and their uses
CA3033327A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
CA3033788A1 (en) 2016-08-17 2018-02-22 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
CN106370765A (zh) * 2016-08-23 2017-02-01 国家烟草质量监督检验中心 一种基于反相色谱飞行时间质谱的喉癌尿液差异代谢物的测定筛选方法
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CN106381310B (zh) * 2016-08-31 2020-02-04 武汉华美生物工程有限公司 一种用t7噬菌体rna聚合酶和t7启动子系统在哺乳动物细胞表达蛋白的方法
JP2019530464A (ja) 2016-10-14 2019-10-24 プレジデント アンド フェローズ オブ ハーバード カレッジ 核酸塩基エディターのaav送達
US20190351071A1 (en) * 2016-11-11 2019-11-21 Dnalite Therapeutics, Inc. Structures and methods for gene therapy
KR101908593B1 (ko) * 2016-12-07 2018-10-16 연세대학교 산학협력단 Cftr 유전자 소실 유도 가이드 rna, cftr 변이를 갖는 t84 세포 및 이의 용도
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
WO2018154418A1 (en) * 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
US11168319B1 (en) * 2017-02-28 2021-11-09 Inari Agriculture Technology, Inc. Plant cell culture
US11407800B2 (en) * 2017-02-28 2022-08-09 Arcturus Therapeutics, Inc. Translatable molecules and synthesis thereof
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
CN107475255B (zh) * 2017-03-21 2021-03-26 河北医科大学第二医院 一种基因载体介导的基于CRISPR/Cas9基因编辑系统的sgRNA及其用途
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 筋萎縮性側索硬化症(als)治療組成物および方法
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
WO2018227432A1 (en) * 2017-06-14 2018-12-20 Wuhan Institute Of Virology, Chinese Academy Of Sciences Cd2-associated protein (cd2ap) and its interactive proteins
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
AU2018352236A1 (en) 2017-10-16 2020-04-23 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
WO2019079347A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. USES OF BASIC EDITORS ADENOSINE
EP3728577A4 (en) 2018-01-22 2021-12-22 Inari Agriculture, Inc. PLANT GENE EDITING SYSTEMS, PROCEDURES AND COMPOSITIONS
AU2019243155A1 (en) * 2018-03-27 2020-10-15 Factor Bioscience Inc. Nucleic acid-based therapeutics
CN108624592B (zh) * 2018-05-24 2021-08-17 苏州大学张家港工业技术研究院 针对DJ-1基因编辑的sgRNA筛选及其载体与应用
CN108949830B (zh) * 2018-08-03 2021-11-26 福州大学 一种在鱼类中实现基因组编辑、精确定点基因敲入的方法
CN109628404B (zh) * 2018-12-18 2020-04-28 浙江大学 猪皮下脂肪前体细胞永生化细胞系的构建方法及用途
WO2020191239A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
CN110200973A (zh) * 2019-06-19 2019-09-06 南京市儿童医院 Dna依赖性蛋白激酶特异性抑制剂在制备防治肾纤维化药物中的用途
WO2021016043A1 (en) 2019-07-19 2021-01-28 Inari Agriculture, Inc. Improved homology dependent repair genome editing
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
CN110699381A (zh) * 2019-09-17 2020-01-17 合肥瑞灵生物科技有限公司 地中海贫血病基因治疗载体构建方法及其用途
WO2021191678A1 (en) 2020-03-23 2021-09-30 Avectas Limited Engineering of dendritic cells for generation of vaccines against sars-cov-2
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
CN113151181B (zh) * 2021-04-14 2022-11-29 中山大学附属第七医院(深圳) 一种脂肪干细胞重编程为神经元的方法及负载该神经元的细胞适应性水凝胶修复脊髓损伤
CN113373214B (zh) * 2021-06-18 2024-03-29 中国人民解放军军事科学院军事医学研究院 Cd30在诊断脑神经相关疾病中的用途
CA3230927A1 (en) 2021-09-10 2023-03-16 Agilent Technologies, Inc. Guide rnas with chemical modification for prime editing
CN114150017B (zh) * 2021-11-30 2023-05-16 长江大学 一种调节猪未成熟睾丸支持细胞乳酸分泌水平的方法及应用
WO2024032678A1 (zh) * 2022-08-11 2024-02-15 益杰立科(上海)生物科技有限公司 一种表观编辑靶点的方法及用途

Family Cites Families (118)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3539465A (en) 1968-10-08 1970-11-10 Ncr Co Encapsulation of hydrophilic liquid-in-oil emulsions
US5843780A (en) 1995-01-20 1998-12-01 Wisconsin Alumni Research Foundation Primate embryonic stem cells
US6743771B2 (en) * 1995-12-29 2004-06-01 Novactyl, Inc. Methods and compositions for controlling protein assembly or aggregation
WO1998030679A1 (en) 1997-01-10 1998-07-16 Life Technologies, Inc. Embryonic stem cell serum replacement
WO1999014346A2 (en) 1997-09-19 1999-03-25 Sequitur, Inc. SENSE mRNA THERAPY
US7621606B2 (en) 2001-08-27 2009-11-24 Advanced Cell Technology, Inc. Trans-differentiation and re-differentiation of somatic cells and production of cells for cell therapies
US20110171185A1 (en) 1999-06-30 2011-07-14 Klimanskaya Irina V Genetically intact induced pluripotent cells or transdifferentiated cells and methods for the production thereof
EP1322656B1 (en) 2000-09-26 2008-01-16 Idera Pharmaceuticals, Inc. Modulation of immunostimulatory activity of immunostimulatory oligonucleotide analogs by positional chemical changes
JPWO2003027277A1 (ja) 2001-09-21 2005-01-06 独立行政法人 科学技術振興機構 再プログラム化因子のスクリーニング方法、該方法によりスクリーニングされた再プログラム化因子および該再プログラム化因子を利用する方法、並びに、未分化融合細胞を分化させる方法および細胞、組織、臓器の製造方法
WO2003033697A1 (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
AU2004253455B2 (en) 2003-06-03 2011-03-03 Eli Lilly And Company Modulation of survivin expression
US7682828B2 (en) 2003-11-26 2010-03-23 Whitehead Institute For Biomedical Research Methods for reprogramming somatic cells
ES2383813T3 (es) 2004-09-08 2012-06-26 Wisconsin Alumni Research Foundation Método de cultivo y cultivo de células madre embrionarias
MX2007002389A (es) 2004-09-08 2009-02-12 Wisconsin Alumni Res Found Cultivo de celulas progenitoras embrionarias humanas.
CA2595695A1 (en) * 2005-01-25 2006-08-03 Cell Therapeutics, Inc. Biologically active protein conjugates comprising (nn[s/t]) peptide repeats and having increased plasma half-life
JP5147692B2 (ja) * 2005-07-13 2013-02-20 ノボ ノルディスク ヘルス ケア アーゲー 治療用タンパク質生成のための宿主細胞タンパク質ノックアウト細胞
CA2615532C (en) * 2005-07-26 2016-06-28 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
EP4174179A3 (en) 2005-08-23 2023-09-27 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
US8476070B2 (en) 2005-08-29 2013-07-02 Technion Research & Development Foundation Limited Media for culturing stem cells
US8129187B2 (en) 2005-12-13 2012-03-06 Kyoto University Somatic cell reprogramming by retroviral vectors encoding Oct3/4. Klf4, c-Myc and Sox2
EP2206724A1 (en) 2005-12-13 2010-07-14 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
US20100168000A1 (en) 2005-12-22 2010-07-01 Peter Kiessling Octanoate-Reduced Human Albumin
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
US20090093551A1 (en) 2006-12-08 2009-04-09 Bhatia Sangeeta N Remotely triggered release from heatable surfaces
US10829733B2 (en) 2007-01-04 2020-11-10 Biolamina Ab Composition and method for enabling proliferation of pluripotent human stem cells
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
AU2008231020B2 (en) 2007-03-23 2013-09-05 Wisconsin Alumni Research Foundation Somatic cell reprogramming
CA2683056C (en) 2007-04-07 2020-03-24 Whitehead Institute For Biomedical Research Reprogramming of somatic cells
JP5558097B2 (ja) 2007-12-10 2014-07-23 国立大学法人京都大学 効率的な核初期化方法
EP2072618A1 (en) 2007-12-14 2009-06-24 Johannes Gutenberg-Universität Mainz Use of RNA for reprogramming somatic cells
ES2690554T3 (es) 2008-03-17 2018-11-21 The Scripps Research Institute Enfoques químicos y genéticos combinados para la generación de células 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
EP2281050B1 (en) * 2008-04-14 2014-04-02 Sangamo BioSciences, Inc. Linear donor constructs for targeted integration
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
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
WO2009147400A1 (en) 2008-06-05 2009-12-10 Iti Scotland Limited 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
EP2326711A2 (en) 2008-08-20 2011-06-01 VIRxSYS Corporation Compositions and methods for generation of pluripotent stem cells
CN105802917A (zh) 2008-10-24 2016-07-27 威斯康星校友研究基金会 通过非病毒重编程获得的多潜能干细胞
EP2192174B1 (en) 2008-11-21 2015-11-11 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Reprogramming cells toward a pluripotent state
US20110023141A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved with parkinson's disease
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
US8219348B2 (en) 2009-01-22 2012-07-10 Johnson Controls Technology Company Method for calibrating and/or correcting a display device having a needle, the needle being able to move in rotation about an axis of rotation
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
US20100272695A1 (en) 2009-04-22 2010-10-28 Alan Agulnick 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
WO2010123501A1 (en) 2009-04-22 2010-10-28 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long rna molecules
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
DK2459231T3 (en) * 2009-07-31 2016-09-05 Ethris Gmbh RNA with a combination of unmodified and modified nucleotides for protein expression
EP2480659A2 (en) * 2009-09-24 2012-08-01 Cellectis Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
AU2010312291A1 (en) * 2009-10-29 2012-06-21 Mcmaster University Generating induced pluripotent stem cells and progenitor cells from fibroblasts
WO2011056971A2 (en) 2009-11-04 2011-05-12 Cellular Dynamics International, Inc. Episomal reprogramming with chemicals
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
PL2510099T3 (pl) 2009-12-07 2018-01-31 The Trustees Of Univ Of Pennsylvania Preparaty RNA zawierające oczyszczony zmodyfikowany RNA do przeprogramowywania komórek
PL2816112T3 (pl) * 2009-12-10 2019-03-29 Regents Of The University Of Minnesota Modyfikacja DNA za pośrednictwem efektorów TAL
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
WO2011110886A1 (en) 2010-03-09 2011-09-15 Biolamina Ab Composition and method for enabling proliferation of pluripotent human stem cells
WO2011130624A2 (en) 2010-04-16 2011-10-20 Immune Disease Institute, Inc. Sustained polypeptide expression from synthetic, modified rnas and uses thereof
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
EP2569424A1 (en) * 2010-05-12 2013-03-20 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
WO2011146121A1 (en) 2010-05-17 2011-11-24 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
CA2802360A1 (en) 2010-06-14 2011-12-22 Iowa State University Research Foundation, Inc. Nuclease activity of tal effector and foki fusion protein
EP2582794B2 (en) * 2010-06-15 2024-04-24 FUJIFILM Cellular Dynamics, Inc. Generation of induced pluripotent stem cells from small volumes of peripheral blood
KR101829488B1 (ko) 2010-08-05 2018-02-14 위스콘신 얼럼나이 리서어치 화운데이션 인간 다분화능 세포 배양을 위한 단순화된 기본 배지
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
WO2012021632A2 (en) * 2010-08-10 2012-02-16 The Johns Hopkins University Generation and use of pluripotent stem cells
GB201014169D0 (en) * 2010-08-25 2010-10-06 Cambridge Entpr Ltd In vitro hepatic differentiation
EP2616540A4 (en) 2010-09-14 2014-02-19 Univ Kyoto METHOD OF EFFICIENTLY ESTABLISHING INDUCED PLURIPOTENT STEM CELLS
AU2011312562B2 (en) * 2010-09-27 2014-10-09 Sangamo Therapeutics, Inc. Methods and compositions for inhibiting viral entry into cells
ES2862955T3 (es) 2010-10-01 2021-10-08 Modernatx Inc Acidos nucleicos manipulados y métodos de uso de los mismos
EP2625278A1 (en) * 2010-10-08 2013-08-14 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
US9499592B2 (en) * 2011-01-26 2016-11-22 President And Fellows Of Harvard College Transcription activator-like effectors
KR20120096395A (ko) * 2011-02-22 2012-08-30 주식회사 툴젠 뉴클레아제에 의해 유전자 변형된 세포를 농축시키는 방법
CN107090470B (zh) * 2011-02-25 2021-09-07 重组股份有限公司 经遗传修饰的动物及其生成方法
EP2683812A4 (en) * 2011-03-07 2014-12-03 Massachusetts Inst Technology METHODS FOR TRANSFECTING CELLS WITH NUCLEIC ACIDS
US8710200B2 (en) 2011-03-31 2014-04-29 Moderna Therapeutics, Inc. Engineered nucleic acids encoding a modified erythropoietin and their expression
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)
US9315788B2 (en) * 2011-04-05 2016-04-19 Cellectis, S.A. Method for the generation of compact TALE-nucleases and uses thereof
WO2013003475A1 (en) 2011-06-27 2013-01-03 Cellscript, Inc. Inhibition of innate immune response
US9394545B2 (en) * 2011-09-21 2016-07-19 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
WO2013052523A1 (en) 2011-10-03 2013-04-11 modeRNA Therapeutics Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
WO2013086008A1 (en) * 2011-12-05 2013-06-13 Factor Bioscience Inc. Methods and products for transfecting cells
US8497124B2 (en) 2011-12-05 2013-07-30 Factor Bioscience Inc. Methods and products for reprogramming cells to a less differentiated state
CN104114572A (zh) 2011-12-16 2014-10-22 现代治疗公司 经修饰的核苷、核苷酸和核酸组合物
CA2859691A1 (en) 2011-12-21 2013-06-27 Moderna Therapeutics, Inc. Methods of increasing the viability or longevity of an organ or organ explant
WO2013102203A1 (en) 2011-12-30 2013-07-04 Cellscript, Inc. MAKING AND USING IN VITRO-SYNTHESIZED ssRNA FOR INTRODUCING INTO MAMMALIAN CELLS TO INDUCE A BIOLOGICAL OR BIOCHEMICAL EFFECT
EP2833920A2 (en) * 2012-04-02 2015-02-11 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and 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
CN112048466A (zh) 2012-04-24 2020-12-08 细胞疗法有限责任公司 从头生成多能细胞
US9738879B2 (en) * 2012-04-27 2017-08-22 Duke University Genetic correction of mutated genes
AU2013263101B2 (en) 2012-05-13 2019-02-14 Allele Biotechnology And 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
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
AU2013289206B2 (en) * 2012-07-11 2018-08-09 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of lysosomal storage diseases
EP2684892A1 (en) * 2012-07-13 2014-01-15 Association Française contre les Myopathies Compositions and methods for duchenne muscular dystrophy gene therapy
DK2906684T3 (da) * 2012-10-10 2020-09-28 Sangamo Therapeutics Inc T-celle-modificerende forbindelser og anvendelser deraf
WO2014071219A1 (en) * 2012-11-01 2014-05-08 Factor Bioscience Inc. Methods and products for expressing proteins in cells
WO2014081855A1 (en) * 2012-11-20 2014-05-30 Universite De Montreal Methods and compositions for muscular dystrophies
EP2929017A4 (en) * 2012-12-05 2016-09-28 Sangamo Biosciences Inc METHODS AND COMPOSITIONS FOR REGULATION OF METABOLIC DISORDERS
HUE056638T2 (hu) 2013-02-22 2022-02-28 Univ Leland Stanford Junior Telomer kiterjesztéssel kapcsolatos gyógyászati alkalmazás
AU2014274840B2 (en) * 2013-06-05 2020-03-12 Duke University RNA-guided gene editing and gene regulation
CA3033788A1 (en) * 2016-08-17 2018-02-22 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof

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