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MX2020006065A - Metodos y productos para la produccion y administracion de acido nucleico. - Google Patents

Metodos y productos para la produccion y administracion de acido nucleico.

Info

Publication number
MX2020006065A
MX2020006065A MX2020006065A MX2020006065A MX2020006065A MX 2020006065 A MX2020006065 A MX 2020006065A MX 2020006065 A MX2020006065 A MX 2020006065A MX 2020006065 A MX2020006065 A MX 2020006065A MX 2020006065 A MX2020006065 A MX 2020006065A
Authority
MX
Mexico
Prior art keywords
methods
nucleic acids
cells
products
therapeutics
Prior art date
Application number
MX2020006065A
Other languages
English (en)
Inventor
Matthew Angel
Christopher Rohde
Original Assignee
Factor Bioscience Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Factor Bioscience Inc filed Critical Factor Bioscience Inc
Publication of MX2020006065A publication Critical patent/MX2020006065A/es

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Abstract

La presente invención se refiere, en parte, a ácidos nucleicos, inclusive ácidos nucleicos que codifican proteínas, agentes terapéuticos y cosméticos que comprenden ácidos nucleicos, métodos para administrar ácidos nucleicos a células, tejidos, órganos y pacientes, métodos para inducir que las células expresen proteínas mediante el uso deácidos nucleicos, kits y dispositivos para transfectar, realizar la edición génica y reprogramar células, y células, organismos y agentes terapéuticos y cosméticos producidos mediante el uso de 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 que las células expresen proteínas, mediante el uso de moléculas sintéticas de ARN, inclusive células presentes in vivo. También se describen agentes terapéuticos que comprenden ácidos nucleicos que codifican proteínas de edición génica.
MX2020006065A 2014-01-31 2016-07-27 Metodos y productos para la produccion y administracion de acido nucleico. MX2020006065A (es)

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Families Citing this family (83)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 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
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
KR102726294B1 (ko) 2014-01-31 2024-11-06 팩터 바이오사이언스 인크. 핵산 제조 및 송달을 위한 방법 및 산물
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
US20160130567A1 (en) 2014-11-02 2016-05-12 Arcturus Therapeutics, Inc. Messenger una molecules and uses thereof
EP3543339A1 (en) 2015-02-13 2019-09-25 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
US10167457B2 (en) 2015-10-23 2019-01-01 President And Fellows Of Harvard College Nucleobase editors and uses thereof
CA3011692A1 (en) 2015-11-23 2017-06-01 The Regents Of The University Of Colorado, A Body Corporate Methods and compositions for reprogramming cells
SG11201808314QA (en) 2016-04-08 2018-10-30 Krystal Biotech Inc Compositions and methods for the treatment of wounds, disorders, and diseases of the skin
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
WO2018003952A1 (ja) * 2016-06-29 2018-01-04 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
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
EP3497214B1 (en) 2016-08-09 2023-06-28 President and Fellows of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
JP2019528284A (ja) * 2016-08-17 2019-10-10 ファクター バイオサイエンス インコーポレイテッド 核酸産物およびその投与方法
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
JP6953537B2 (ja) * 2016-10-14 2021-10-27 ネオマトリクス・セラピューティクス・インコーポレイテッド 生物活性が改善され好中球エラスターゼ分解に対する感受性が低下した、フィブロネクチンに由来するペプチド
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
US11464836B2 (en) 2016-12-08 2022-10-11 Curevac Ag RNA for treatment or prophylaxis of a liver disease
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
BR112019019655A2 (pt) 2017-03-23 2020-04-22 Harvard College editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico
EP3600379B1 (en) 2017-03-31 2022-12-21 Accanis Biotech F&E GmbH & Co KG Prevention and treatment of non-melanoma skin cancer (nmsc)
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
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
BR112019026625A2 (pt) 2017-06-15 2020-06-30 The Regents Of The University Of California inserções de dna não viral direcionadas
US11732274B2 (en) 2017-07-28 2023-08-22 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
US11319532B2 (en) 2017-08-30 2022-05-03 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
AU2018352592C1 (en) 2017-10-16 2025-09-25 Beam Therapeutics, 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
EP3775202A4 (en) * 2018-03-27 2022-04-06 Factor Bioscience Inc. NUCLEIC ACID BASED THERAPEUTICS
JP2021528041A (ja) 2018-04-05 2021-10-21 アジトラ インコーポレーテッド 組換え微生物を用いて皮膚疾患を処置するための方法および組成物
EP3775236A1 (en) 2018-04-12 2021-02-17 Krystal Biotech, LLC Compositions and methods for the treatment of autosomal recessive congenital ichthyosis
JP7602999B2 (ja) * 2018-04-27 2024-12-19 クリスタル バイオテック インコーポレイテッド 審美的用途のための美容用タンパク質をコードする組換え核酸
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
CA3109592A1 (en) 2018-08-23 2020-02-27 Sangamo Therapeutics, Inc. Engineered target specific base editors
JP7562515B2 (ja) 2018-09-24 2024-10-07 クリスタル バイオテック インコーポレイテッド ネザートン症候群の治療のための組成物及び方法
CA3266235A1 (en) * 2018-09-26 2025-11-29 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
WO2020091137A1 (en) * 2018-10-31 2020-05-07 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
KR20210124277A (ko) 2019-02-08 2021-10-14 크리스탈 바이오테크, 인크. Cftr 폴리펩타이드를 전달하기 위한 조성물 및 방법
DK3930676T3 (en) * 2019-03-01 2022-10-31 Flagship Pioneering Innovations Vi Llc Polyribonucleotides and cosmetic uses thereof
CN114127285B (zh) 2019-03-19 2024-09-10 布罗德研究所股份有限公司 编辑核苷酸序列的方法和组合物
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US20210000910A1 (en) 2019-07-03 2021-01-07 Jysk Skin Solutions Pte Ltd Topical compositions
CN114450265B (zh) 2019-07-03 2024-12-24 菲克特生物科学股份有限公司 阳离子脂质及其用途
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
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
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
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
CA3213789A1 (en) 2021-04-02 2022-10-06 Krystal Biotech, Inc. Viral vectors for cancer therapy
CN113603739B (zh) * 2021-08-27 2024-08-23 上海兆维科技发展有限公司 一种加帽类似物及其应用
IL311219A (en) * 2021-09-08 2024-05-01 Flagship Pioneering Innovations Vi Llc Methods and compositions for genome modulation
PE20241173A1 (es) 2021-10-14 2024-05-28 Arsenal Biosciences Inc Celulas inmunitarias que tienen arnhc coexpresados y sistemas de compuerta logica
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
MX2024008082A (es) * 2022-01-05 2024-07-15 Helix Nanotechnologies Inc Composiciones que comprenden la preprosecuencia del factor-alfa y sus usos.
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
KR20240167444A (ko) * 2022-03-30 2024-11-26 야마사 쇼유 가부시키가이샤 피브릴린 산생 촉진제, 화장료 조성물 또는 피부 외용제, 경구 투여제 및 엘라스틴 섬유 산생 촉진제
US12083204B2 (en) 2022-06-02 2024-09-10 L'oreal Topical composition for homeostatic delivery of nitric oxide and uses thereof
WO2024035146A1 (ko) 2022-08-10 2024-02-15 한화솔루션 주식회사 이소시아네이트 조성물의 제조 방법 및 이소시아네이트 조성물
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
WO2024214050A1 (en) * 2023-04-11 2024-10-17 Eliksa Therapeutics, Inc. Methods of increasing therapeutic protein levels and/or improving cellular function with amniotic fluid compositions
WO2025024500A1 (en) * 2023-07-25 2025-01-30 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

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
AU9319398A (en) 1997-09-19 1999-04-05 Sequitur, Inc. Sense mrna therapy
EP1187653B1 (en) * 1999-06-04 2010-03-31 Georgia Tech Research Corporation Devices 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
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
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
WO2002026757A2 (en) 2000-09-26 2002-04-04 Hybridon, Inc. Modulation of immunostimulatory activity of immunostimulatory oligonucleotide analogs by positional chemical changes
US7189759B2 (en) 2001-05-23 2007-03-13 Medicis Pharmaceutical Corporation Compositions for the treatment of pigmentation disorders and methods for their manufacture
US20050130144A1 (en) 2001-09-21 2005-06-16 Norio Nakatsuji Method of screening reprogramming factor, reprogramming factor screened by the method, method of using the reprogramming factor, method of differentiating undifferentiated fused cells and method of constructing cell, tissues and organs
JP2005506074A (ja) 2001-10-18 2005-03-03 イクシオン・バイオテクノロジー・インコーポレーテッド 肝臓の幹細胞および前駆細胞の膵臓機能細胞への転換
US7276489B2 (en) 2002-10-24 2007-10-02 Idera Pharmaceuticals, Inc. Modulation of immunostimulatory properties of oligonucleotide-based compounds by optimal presentation of 5′ ends
GB0202149D0 (en) 2002-01-30 2002-03-20 Univ Edinburgh Pluripotency determining factors and uses thereof
US20030186249A1 (en) * 2002-04-01 2003-10-02 Zairen Sun Human TARPP genes and polypeptides
JP3785508B2 (ja) 2002-04-15 2006-06-14 学校法人慶應義塾 遺伝子治療における免疫応答を解析できる実験モデルマウス
US7737182B2 (en) * 2002-08-09 2010-06-15 Taisho Pharmaceutical Co., Ltd. Pharmaceuticals for xerosis
US7541344B2 (en) 2003-06-03 2009-06-02 Eli Lilly And Company Modulation of survivin expression
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
EP1691780A2 (en) 2003-11-21 2006-08-23 Alza Corporation Gene delivery mediated by liposome-dna complex with cleavable peg surface modification
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
WO2006029198A2 (en) 2004-09-08 2006-03-16 Wisconsin Alumni Research Foundation Culturing human embryonic stem cells
WO2007014275A2 (en) 2005-07-26 2007-02-01 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
US8323666B2 (en) 2005-08-01 2012-12-04 Allergan, Inc. Botulinum toxin compositions
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
EP4174179B1 (en) 2005-08-23 2025-05-07 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
EP3354723B1 (en) 2005-08-29 2023-12-13 Technion Research & Development Foundation Ltd. 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
US8278104B2 (en) 2005-12-13 2012-10-02 Kyoto University Induced pluripotent stem cells produced with Oct3/4, Klf4 and Sox2
EP1970446B1 (en) 2005-12-13 2011-08-03 Kyoto University Nuclear reprogramming factor
KR20080078010A (ko) 2005-12-22 2008-08-26 체에스엘 베링 게엠베하 옥타노에이트-감소된 사람 알부민
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
EP2099496A2 (en) 2006-12-08 2009-09-16 Massachusetts Institute of Technology Delivery of nanoparticles and/or agents to cells
CN101200758A (zh) 2006-12-15 2008-06-18 华中科技大学 一种检测col7a1基因突变的方法及其用途
US10829733B2 (en) 2007-01-04 2020-11-10 Biolamina Ab Composition and method for enabling proliferation of pluripotent human stem cells
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
SG193652A1 (en) 2007-03-23 2013-10-30 Wisconsin Alumni Res Found Somatic cell reprogramming
EP3878949B1 (en) 2007-04-07 2025-06-04 Whitehead Institute for Biomedical Research Reprogramming of somatic cells
WO2009046739A1 (en) * 2007-10-09 2009-04-16 Curevac Gmbh Composition for treating prostate cancer (pca)
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
WO2009117439A2 (en) 2008-03-17 2009-09-24 The Scripps Research Institute Combined chemical and genetic approaches for generation of induced pluripotent stem cells
US20110045001A1 (en) 2008-03-28 2011-02-24 Biontex Laboratories Gmbh Transfection results of non-viral gene delivery systems by influencing of the innate immune system
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
CA2726990C (en) 2008-06-04 2020-09-08 Cellular Dynamics International, Inc. Methods for the production of ips cells using extra-chromosomal vectors
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
US20110263015A1 (en) 2008-08-20 2011-10-27 Virxsys Corporation Compositions and methods for generation of pluripotent stem cells
US8268620B2 (en) 2008-10-24 2012-09-18 Wisconsin Alumni Research Foundation OCT4 and SOX2 with SV40 T antigen produce pluripotent stem cells from primate somatic cells
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
WO2010093655A2 (en) 2009-02-10 2010-08-19 University Of Dayton Enhanced method for producing stem-like cells from somatic cells
US10894944B2 (en) 2009-04-10 2021-01-19 Monash University Cell culture media
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
EP3904505A1 (en) 2009-04-22 2021-11-03 Viacyte, Inc. Cell compositions derived from dedifferentiated reprogrammed cells
AU2009344887A1 (en) * 2009-04-22 2011-11-10 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
JP5944311B2 (ja) * 2009-06-16 2016-07-05 クルナ・インコーポレーテッド コラーゲン遺伝子に対する天然アンチセンス転写物の抑制によるコラーゲン遺伝子関連疾患の治療
DK2459231T3 (en) 2009-07-31 2016-09-05 Ethris Gmbh RNA with a combination of unmodified and modified nucleotides for protein expression
AU2010315166B2 (en) 2009-11-04 2015-01-22 FUJIFILM Cellular Dynamics, Inc. Episomal reprogramming with chemicals
EP2499239A1 (en) 2009-11-11 2012-09-19 Sanford-Burnham Medical Research Institute METHOD FOR GENERATION AND REGULATION OF iPS CELLS AND COMPOSITIONS THEREOF
KR102171849B1 (ko) 2009-12-07 2020-10-30 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 세포 리프로그래밍을 위한 정제된 변형 ｒｎａ를 포함하는 ｒｎａ 제제
US20130189741A1 (en) 2009-12-07 2013-07-25 Cellscript, Inc. Compositions and methods for reprogramming mammalian cells
TR201815882T4 (tr) 2009-12-10 2018-11-21 Univ Iowa State Res Found Inc Tal efektörü aracılı dna modifikasyonu.
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
US8962331B2 (en) 2010-02-01 2015-02-24 The Board Of Trustees Of The Leland Stanford Junior University Method of making induced pluripotent stem cell from adipose stem cells using minicircle DNA vectors
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
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
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
EP2392208B1 (en) 2010-06-07 2016-05-04 Helmholtz Zentrum München Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Fusion proteins comprising a DNA-binding domain of a Tal effector protein and a non-specific cleavage domain of a restriction nuclease and their use
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
EP2600901B1 (en) 2010-08-06 2019-03-27 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
US9447408B2 (en) 2010-09-14 2016-09-20 Kyoto University Method of efficiently establishing induced pluripotent stem cells
CN104531812A (zh) 2010-10-01 2015-04-22 现代治疗公司 设计核酸及其使用方法
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
WO2012122318A2 (en) 2011-03-07 2012-09-13 Massachusetts Institute Of Technology Methods for transfecting cells with nucleic acids
WO2012131090A1 (en) * 2011-03-31 2012-10-04 Galderma Research & Development Method for treatment of xeroderma pigmentosum
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)
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
EP2732029B1 (en) 2011-07-11 2019-01-16 FUJIFILM Cellular Dynamics, Inc. Methods for cell reprogramming and genome engineering
MX351565B (es) 2011-08-03 2017-10-19 Phoenix Tissue Repair Inc Colageno 7 y metodos relacionados.
BR112014007852B1 (pt) 2011-10-03 2021-11-03 Moderna Therapeutics, Inc Polinucleotídeo isolado modificado e composição farmacêutica
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
JP6073916B2 (ja) 2011-12-05 2017-02-01 ファクター バイオサイエンス インコーポレイテッド 細胞に形質移入するための方法および製品
US8497124B2 (en) 2011-12-05 2013-07-30 Factor Bioscience Inc. Methods and products for reprogramming cells to a less differentiated state
PL2791160T3 (pl) * 2011-12-16 2022-06-20 Modernatx, Inc. Kompozycje zmodyfikowanego mrna
JP2015510495A (ja) 2011-12-21 2015-04-09 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 器官または器官移植片の生存可能性または寿命を延長する方法
EP2798064B1 (en) 2011-12-30 2016-08-31 Cellscript, Llc Making and using in vitro-synthesized ssrna for introducing into mammalian cells to induce a biological or biochemical effect
HK1206779A1 (en) * 2012-04-02 2016-01-15 Modernatx, Inc. Modified polynucleotides for the production of nuclear proteins
WO2013163296A1 (en) 2012-04-24 2013-10-31 The Brigham And Women's Hospital, Inc. 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
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
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
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
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
EP3567104A1 (en) * 2013-03-01 2019-11-13 Regents of the University of Minnesota Talen-based gene correction
SG11201601939VA (en) * 2013-09-16 2016-04-28 Agency Science Tech & Res Method
KR102726294B1 (ko) 2014-01-31 2024-11-06 팩터 바이오사이언스 인크. 핵산 제조 및 송달을 위한 방법 및 산물

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