FI3690056T3 - Menetelmiä ja tuotteita nukleiinihapon valmistamiseksi ja viemiseksi - Google Patents

Menetelmiä ja tuotteita nukleiinihapon valmistamiseksi ja viemiseksi Download PDF

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Publication number
FI3690056T3
FI3690056T3 FIEP20161924.4T FI20161924T FI3690056T3 FI 3690056 T3 FI3690056 T3 FI 3690056T3 FI 20161924 T FI20161924 T FI 20161924T FI 3690056 T3 FI3690056 T3 FI 3690056T3
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gene
composition
protein
rna molecule
used according
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FIEP20161924.4T
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English (en)
Finnish (fi)
Swedish (sv)
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Matthew Angel
Christopher Rohde
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Factor Bioscience Inc
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Families Citing this family (85)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
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
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
KR102415811B1 (ko) 2014-01-31 2022-07-04 팩터 바이오사이언스 인크. 핵산 제조 및 송달을 위한 방법 및 산물
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016070166A2 (en) 2014-11-02 2016-05-06 Arcturus Therapeutics, Inc. Messenger una molecules and uses thereof
US11241505B2 (en) * 2015-02-13 2022-02-08 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
EP3384038B1 (en) * 2015-11-23 2024-07-24 The Regents of The University of Colorado, A Body Corporate Methods and compositions for reprogramming cells
ES2796488T3 (es) * 2016-04-08 2020-11-27 Krystal Biotech Inc Composiciones para el uso en métodos para el tratamiento de heridas, trastornos y enfermedades de la piel
EP3241905A1 (en) * 2016-05-06 2017-11-08 Miltenyi Biotec GmbH Method for introducing nucleic acids into a cell
WO2017197141A2 (en) * 2016-05-13 2017-11-16 Sangamo Therapeutics, Inc. Targeted treatment of androgenic alopecia
JP7183041B2 (ja) * 2016-06-29 2022-12-05 センジュ ユーエスエー、インコーポレイテッド 経皮薬物送達システムおよびその使用方法
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
IL264439B2 (en) * 2016-08-17 2024-08-01 Factor Bioscience Inc non-viral, cell-free composition comprising a synthetic messenger RNA (MRNA) encoding a gene-editing protein for use in treating cancer, and a synthetic RNA encoding a gene-editing protein for use in treatment
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CN110023325B (zh) * 2016-10-14 2023-07-04 尼优美特里克斯医疗有限公司 具有提高的生物活性和降低的对嗜中性粒细胞弹性蛋白酶降解易感性的来源于纤连蛋白的肽
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
WO2018104538A1 (en) * 2016-12-08 2018-06-14 Curevac Ag Rna for treatment or prophylaxis of a liver disease
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
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
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
CN110636854A (zh) 2017-03-31 2019-12-31 阿卡尼斯生物技术F&E有限责任两合公司 非黑素瘤皮肤癌(nmsc)的预防和治疗
CN107400635A (zh) * 2017-04-27 2017-11-28 广州弘宝元生物科技有限公司 导入α‑MSH多肽的粘红酵母重组菌株及其制备方法和应用
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
MY200418A (en) 2017-06-15 2023-12-25 Univ California Targeted non-viral dna insertions
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
BR102017016440A2 (pt) 2017-07-31 2019-03-19 Universidade Federal Do Rio Grande Do Sul Composição para terapia gênica do sistema nervoso central, processo de obtenção e uso da mesma
EP3437650A1 (en) * 2017-07-31 2019-02-06 Accanis Biotech F&E GmbH & Co KG Treatment of local skin hypotrophy conditions
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
EP3678640A4 (en) 2017-09-05 2021-06-09 Azitra, Inc. METHODS AND COMPOSITIONS FOR TREATMENT OF INFLAMMATORY SKIN DISEASES WITH RECOMBINANT MICRO-ORGANISMS
CA3078631A1 (en) * 2017-10-11 2019-04-18 Jacob M. Waugh Methods and compositions for topical delivery
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
SG11202003798TA (en) 2017-10-27 2020-05-28 Univ California Targeted replacement of endogenous t cell receptors
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3543351B1 (en) 2018-03-19 2022-08-10 Ricoh Company, Ltd. Nucleic acid sample-contained container, method for producing nucleic acid sample-contained container, and nucleic acid sample
CA3095125A1 (en) * 2018-03-27 2019-10-03 Factor Bioscience Inc. Nucleic acid-based therapeutics
KR102958724B1 (ko) 2018-04-05 2026-05-06 아지트라 인코포레이티드 재조합 미생물을 사용하여 피부 질환을 치료하기 위한 방법 및 조성물
EP3775236A1 (en) 2018-04-12 2021-02-17 Krystal Biotech, LLC Compositions and methods for the treatment of autosomal recessive congenital ichthyosis
MX2020011257A (es) 2018-04-27 2021-01-15 Krystal Biotech Inc Ácidos nucleicos recombinantes que codifican una o más proteínas cosméticas para usos estéticos.
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
JP7541508B2 (ja) 2018-08-23 2024-08-28 サンガモ セラピューティクス, インコーポレイテッド 操作された標的特異的な塩基エディター
AU2019346549B2 (en) 2018-09-24 2024-09-12 Krystal Biotech, Inc. Compositions and methods for the treatment of Netherton Syndrome
EP3856914A1 (en) 2018-09-26 2021-08-04 Krystal Biotech, Inc. Compositions and methods for the treatment of skin diseases
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12174174B2 (en) * 2018-10-31 2024-12-24 National Cancer Center Composition comprising material for regulating OCT4 modification to repress stemness
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
CN113454105A (zh) 2019-02-08 2021-09-28 克里斯托生物技术股份有限公司 用于递送cftr多肽的组合物和方法
KR102901623B1 (ko) * 2019-03-01 2025-12-18 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 폴리리보뉴클레오티드 및 이의 미용적 용도
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CA3145425A1 (en) 2019-07-03 2021-01-07 Factor Bioscience Inc. Cationic lipids and uses thereof
US20210000910A1 (en) 2019-07-03 2021-01-07 Jysk Skin Solutions Pte Ltd Topical compositions
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
CN110467667B (zh) * 2019-09-03 2020-11-24 中国科学院理化技术研究所 一种抗肿瘤活性肽及其应用
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
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
IL307350A (en) 2021-04-02 2023-11-01 Krystal Biotech Inc Viral vectors for cancer treatment
CN118978560A (zh) * 2021-08-27 2024-11-19 上海兆维科技发展有限公司 一种加帽类似物及其应用
MX2024002930A (es) * 2021-09-08 2024-05-29 Flagship Pioneering Innovations Vi Llc Metodos y composiciones para modular un genoma.
MX2024003887A (es) 2021-10-14 2024-07-09 Arsenal Biosciences Inc Células inmunitarias que tienen arnch coespresados y sistemas de compuerta lógica.
CN114150022B (zh) * 2021-12-06 2023-08-22 中国科学院精密测量科学与技术创新研究院 一种基于植物微纳结构的生化分子细胞递送方法及应用
JP7271026B1 (ja) * 2021-12-28 2023-05-11 株式会社Hyperion Drug Discovery 遺伝子導入細胞の免疫応答を回避させるための剤
EP4460509A1 (en) * 2022-01-05 2024-11-13 Helix Nanotechnologies Inc Compositions comprising alpha-factor prepro sequence and uses thereof
CN114480365B (zh) * 2022-01-18 2024-05-28 南昌大学 一种高分子-酶-无机杂化纳米花及其制备方法与其在降解食用油中真菌毒素的应用
JPWO2023191016A1 (enExample) * 2022-03-30 2023-10-05
US12083204B2 (en) 2022-06-02 2024-09-10 L'oreal Topical composition for homeostatic delivery of nitric oxide and uses thereof
JP2025530650A (ja) 2022-08-10 2025-09-17 ハンワ ソリューションズ コーポレイション イソシアネート組成物の製造方法およびイソシアネート組成物
EP4374870A1 (en) * 2022-11-22 2024-05-29 Eberhard Karls Universität Tübingen Medizinische Fakultät Codon-optimized oligonucleotide for induction of elastin de-novo synthesis in mammals
WO2024158256A1 (ko) * 2023-01-27 2024-08-02 주식회사 차메디텍 니도겐을 포함하는 피부 상태 개선, 피부 손상 치료 또는 줄기세포능 유지용 조성물
CN116474120A (zh) * 2023-04-03 2023-07-25 臻赫医药(杭州)有限公司 重编程因子抗衰老mRNA组合物、制备方法及应用
US20260232737A1 (en) * 2023-04-11 2026-08-13 Eliksa Therapeutics, Inc. Methods of increasing therapeutic protein levels and/or improving cellular function with amniotic fluid compositions
EP4750515A1 (en) * 2023-07-25 2026-06-03 PassPort Technologies, Inc. Methods and systems for transdermal immunotherapy
CN117683116B (zh) * 2023-10-17 2025-08-15 百世美生物技术(浙江)有限公司 一种重组人源弹性蛋白及其制备方法与应用
CN117904149A (zh) * 2023-10-25 2024-04-19 中国医学科学院北京协和医院 一种表达酪氨酸酶的mRNA及用途
US20260000702A1 (en) 2024-06-26 2026-01-01 Flagship Pioneering Innovations Vii, Llc Therapeutic circular dna forms

Family Cites Families (126)

* 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
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
US6602693B1 (en) 1996-07-03 2003-08-05 Clear Solutions Biotech, Inc. Gene encoding hyaluronan synthase
EP0955999B1 (en) 1996-08-19 2001-12-05 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Novel liposome complexes for increased systemic delivery
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
TW520297B (en) 1996-10-11 2003-02-11 Sequus Pharm Inc Fusogenic liposome composition and method
JP2001508302A (ja) 1997-01-10 2001-06-26 ライフ テクノロジーズ,インコーポレイテッド 胚性幹細胞血清置換
US6316260B1 (en) 1997-08-22 2001-11-13 Bernina Biosystems Gmbh Tetraether lipid derivatives and liposomes and lipid agglomerates containing tetraether lipid derivatives, and use thereof
EP1021549A2 (en) 1997-09-19 2000-07-26 Sequitur, Inc. SENSE mRNA THERAPY
US7390780B2 (en) 1999-04-23 2008-06-24 Alza Corporation Gene delivery mediated by liposome-DNA complex with cleavable peg surface modification
WO2000074763A2 (en) 1999-06-04 2000-12-14 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
US20020064876A1 (en) * 1999-12-28 2002-05-30 Kyonggeun Yoon Novel gene therapy methods for the treatment of skin disorders
US6595947B1 (en) 2000-05-22 2003-07-22 Becton, Dickinson And Company Topical delivery of vaccines
EP2292632A3 (en) 2000-09-26 2012-07-25 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
WO2003027277A1 (en) 2001-09-21 2003-04-03 Japan Science And Technology Corporation Method of screening reporgramming factor, reprogramming factor screened by the method, method of using the reprogramming factor, method of differentiating undifferentiated fused cells and method of constructing cell, tissues and organs
US20050053588A1 (en) 2001-10-18 2005-03-10 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
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
JP2005046073A (ja) * 2003-07-29 2005-02-24 National Institute Of Advanced Industrial & Technology 修飾核酸を用いるrna転写および/または増幅方法
US7682828B2 (en) 2003-11-26 2010-03-23 Whitehead Institute For Biomedical Research Methods for reprogramming somatic cells
BRPI0514778B1 (pt) 2004-09-08 2018-07-10 Wisconsin Alumni Research Foundation Meio para cultivar células tronco embrionárias
BRPI0514641A (pt) 2004-09-08 2008-06-17 Wisconsin Alumni Res Found cultivação das células tronco embriÈnica humana
EP1913149A4 (en) 2005-07-26 2009-08-05 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
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
WO2007026353A2 (en) 2005-08-29 2007-03-08 Technion Research & Development Foundation Ltd. Media for culturing stem cells
US8278104B2 (en) 2005-12-13 2012-10-02 Kyoto University Induced pluripotent stem cells produced with Oct3/4, Klf4 and Sox2
EP2206724A1 (en) 2005-12-13 2010-07-14 Kyoto University Nuclear reprogramming factor
US8129187B2 (en) 2005-12-13 2012-03-06 Kyoto University Somatic cell reprogramming by retroviral vectors encoding Oct3/4. Klf4, c-Myc and Sox2
WO2007079886A1 (de) 2005-12-22 2007-07-19 Csl Behring Gmbh Oktanoatreduziertes 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
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
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
DK3399025T3 (da) 2007-03-23 2025-06-16 Wisconsin Alumni Res Found Omprogrammering af somatiske celler
MX348010B (es) 2007-04-07 2017-05-23 Whitehead Inst Biomedical Res Reprogramacion de celulas somaticas.
WO2009046739A1 (en) * 2007-10-09 2009-04-16 Curevac Gmbh Composition for treating prostate cancer (pca)
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
JP5951254B2 (ja) 2008-03-17 2016-07-13 ザ スクリプス リサーチ インスティテュート 人工多能性幹細胞を作製するための化学的手法と遺伝的手法の組み合わせ法
US20110045001A1 (en) 2008-03-28 2011-02-24 Biontex Laboratories Gmbh Transfection results of non-viral gene delivery systems by influencing of the innate immune system
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
ES2587395T3 (es) 2008-06-04 2016-10-24 Cellular Dynamics International, Inc. Procedimientos para la producción de células IPS usando un enfoque no vírico
WO2009147400A1 (en) 2008-06-05 2009-12-10 Iti Scotland Limited Stem cell culture media and methods
WO2010008486A2 (en) 2008-06-24 2010-01-21 Parkinsons Institute Pluripotent cell lines and methods of use thereof
US20100184033A1 (en) 2008-07-16 2010-07-22 West Michael D Methods to accelerate the isolation of novel cell strains from pluripotent stem cells and cells obtained thereby
EP2151248A1 (en) 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
EP2326711A2 (en) 2008-08-20 2011-06-01 VIRxSYS Corporation Compositions and methods for generation of pluripotent stem cells
EP3450545B1 (en) 2008-10-24 2023-08-23 Wisconsin Alumni Research Foundation Pluripotent stem cells obtained by non-viral reprogramming
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
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
WO2010123501A1 (en) 2009-04-22 2010-10-28 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long rna molecules
ES2849181T3 (es) 2009-04-22 2021-08-16 Viacyte Inc Composiciones celulares derivadas de células reprogramadas desdiferenciadas
US10837020B2 (en) 2009-04-22 2020-11-17 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long RNA molecules
JP2012524777A (ja) * 2009-04-22 2012-10-18 マサチューセッツ インスティテュート オブ テクノロジー 長いrna分子の反復送達を可能にする自然免疫抑制
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
EA022786B1 (ru) 2009-07-31 2016-03-31 Этрис Гмбх Рнк с комбинацией из немодифицированных и модифицированных нуклеотидов для экспрессии белков
KR101775926B1 (ko) 2009-11-04 2017-09-07 셀룰러 다이내믹스 인터내셔널, 인코포레이티드 화학 물질을 이용한 에피솜 재프로그래밍
EP2499239A1 (en) 2009-11-11 2012-09-19 Sanford-Burnham Medical Research Institute METHOD FOR GENERATION AND REGULATION OF iPS CELLS AND COMPOSITIONS THEREOF
ES2769129T3 (es) 2009-12-07 2020-06-24 Univ Pennsylvania Preparaciones de ARN que comprenden ARN modificado purificado para reprogramar células
US20130189741A1 (en) 2009-12-07 2013-07-25 Cellscript, Inc. Compositions and methods for reprogramming mammalian cells
PT2816112T (pt) 2009-12-10 2018-11-20 Univ Iowa State Res Found Inc Modificação do adn modificada pelo efector 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
US20140194482A1 (en) 2010-02-02 2014-07-10 Scioderm, Inc. Compositions and methods of treatment of inflammatory skin conditions using allantoin
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
US8048675B1 (en) 2010-05-12 2011-11-01 Ipierian, Inc. Integration-free human induced pluripotent stem cells from blood
EP2569424A1 (en) 2010-05-12 2013-03-20 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
AU2011256838B2 (en) 2010-05-17 2014-10-09 Sangamo Therapeutics, 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
CN108531444A (zh) 2010-08-05 2018-09-14 威斯康星校友研究基金会 用于人多能细胞培养的简化基础培养基
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
US9447408B2 (en) 2010-09-14 2016-09-20 Kyoto University Method of efficiently establishing induced pluripotent stem cells
EP2857499A1 (en) 2010-10-01 2015-04-08 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
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
AU2012225497A1 (en) 2011-03-07 2013-10-24 Massachusetts Institute Of Technology Methods for transfecting cells with nucleic acids
WO2012131090A1 (en) 2011-03-31 2012-10-04 Galderma Research & Development Method for treatment of xeroderma pigmentosum
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
US10086043B2 (en) 2011-04-03 2018-10-02 The General Hospital Corporation Efficient protein expression in vivo using modified RNA (MOD-RNA)
WO2012174224A2 (en) * 2011-06-17 2012-12-20 Calando Pharmaceuticals, Inc. Methods for administering nucleic acid-based therapeutics
WO2012176015A1 (en) 2011-06-24 2012-12-27 Leo Pharma A/S Methods for treating uv-damaged skin and scc tumors and for removing tattoos with topical ingenol mebutate
US9862926B2 (en) 2011-06-27 2018-01-09 Cellscript, Llc. Inhibition of innate immune response
WO2013009825A1 (en) * 2011-07-11 2013-01-17 Cellular Dynamics International, Inc. Methods for cell reprogramming and genome engineering
CN103842517A (zh) 2011-08-03 2014-06-04 莲花组织修复公司 胶原蛋白7及相关方法
PT3682905T (pt) 2011-10-03 2022-04-07 Modernatx Inc Nucleósidos, nucleótidos e ácidos nucleicos modificados e respetivas utilizações
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
JP2013086009A (ja) 2011-10-17 2013-05-13 Yamamoto Co Ltd 自動精米装置
EP3260140B1 (en) 2011-12-05 2021-02-03 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
ES2923757T3 (es) 2011-12-16 2022-09-30 Modernatx Inc Composiciones de ARNm modificado
AU2012358384A1 (en) 2011-12-21 2014-07-31 Moderna Therapeutics, Inc. Methods of increasing the viability or longevity of an organ or organ explant
DK3421601T3 (da) 2011-12-30 2020-02-24 Cellscript Llc Fremstilling og anvendelse af in-vitro-syntetiseret ssRNA til indføring i pattedyrceller med henblik på at inducere en biologisk eller biokemisk effekt
WO2013151664A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
AU2013251649B2 (en) 2012-04-24 2019-04-04 Vcell Therapeutics, Inc. Generating pluripotent cells de novo
CN104520424B (zh) 2012-05-13 2019-06-28 美国绿阳生物技术及医药公司 使用合成的信使rna无饲养细胞地衍生人类诱导性多能干细胞
US10119150B2 (en) 2012-05-13 2018-11-06 Allele Biotechnology & Pharmaceuticals, Inc. Feeder-free Derivation of human-induced pluripotent stem cells with synthetic messenger RNA
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
WO2014015314A1 (en) 2012-07-19 2014-01-23 University Of Southern California Recombinant c7 and methods of use
KR101979746B1 (ko) * 2012-09-13 2019-05-17 (주)아모레퍼시픽 전기 천공 장치
SG10201707569YA (en) * 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
US10525075B2 (en) 2013-02-22 2020-01-07 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
US20160220613A1 (en) * 2013-09-16 2016-08-04 Agency For Science, Technology And Research Method
KR102415811B1 (ko) 2014-01-31 2022-07-04 팩터 바이오사이언스 인크. 핵산 제조 및 송달을 위한 방법 및 산물

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