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.

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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.
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Families Citing this family (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3613852A3 (en) 2011-07-22 2020-04-22 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
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
US10077453B2 (en) 2014-07-30 2018-09-18 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
WO2016131052A1 (en) * 2015-02-13 2016-08-18 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
IL294014B1 (en) 2015-10-23 2024-03-01 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
EP3377637B1 (en) * 2016-04-08 2020-03-18 Krystal Biotech, LLC Compositions for use in 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
AU2017306676B2 (en) 2016-08-03 2024-02-22 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
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
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CN110214180A (zh) 2016-10-14 2019-09-06 哈佛大学的校长及成员们 核碱基编辑器的aav递送
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
JP2020501545A (ja) * 2016-12-08 2020-01-23 キュアバック アーゲー 肝疾患の処置または予防のためのrna
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 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 シトシンからグアニンへの塩基編集因子
KR20190130613A (ko) 2017-03-23 2019-11-22 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
US20200038487A1 (en) 2017-03-31 2020-02-06 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
US20200362355A1 (en) 2017-06-15 2020-11-19 The Regents Of The University Of California Targeted non-viral dna insertions
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)
EP3437650A1 (en) * 2017-07-31 2019-02-06 Accanis Biotech F&E GmbH & Co KG Treatment of local skin hypotrophy conditions
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
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
AU2018328119A1 (en) * 2017-09-05 2020-04-02 Azitra Inc Methods and compositions for treating inflammatory skin disease with recombinant microorganisms
WO2019075263A2 (en) * 2017-10-11 2019-04-18 Illustris Pharmaceuticals, Inc. METHODS AND COMPOSITIONS WITH TOPICAL ADMINISTRATION
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
AU2018355587B2 (en) 2017-10-27 2023-02-02 The Regents Of The University Of California Targeted replacement of endogenous T cell receptors
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
AU2019249249A1 (en) * 2018-04-05 2020-10-22 Azitra Inc Methods and compositions for treating skin disease with recombinant microorganisms
AU2019252658B2 (en) 2018-04-12 2023-09-14 Krystal Biotech, Inc. 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.
IL280951B1 (en) 2018-08-23 2024-04-01 Sangamo Therapeutics Inc Engineered target-specific base editors
AU2019346549A1 (en) 2018-09-24 2021-04-15 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
US20220034872A1 (en) * 2018-10-31 2022-02-03 National Cancer Center Composition comprising material for regulating oct4 modification to repress stemness
EP3921334A1 (en) 2019-02-08 2021-12-15 Krystal Biotech, Inc. Compositions and methods for delivering cftr polypeptides
EP3942040A1 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
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
MX2022014008A (es) 2020-05-08 2023-02-09 Broad Inst Inc Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo.
CN114717229A (zh) * 2021-01-05 2022-07-08 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
EP4314028A1 (en) 2021-04-02 2024-02-07 Krystal Biotech, Inc. Viral vectors for cancer therapy
WO2023039407A2 (en) * 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
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
CA3238793A1 (en) * 2022-01-05 2023-07-13 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
WO2023191016A1 (ja) * 2022-03-30 2023-10-05 ヤマサ醤油株式会社 フィブリリン産生促進剤、化粧料組成物又は皮膚外用剤、経口投与剤、及びエラスチン線維産生促進剤

Family Cites Families (121)

* 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
US6770291B2 (en) 1996-08-30 2004-08-03 The United States Of America As Represented By The Department Of Health And Human Services Liposome complexes for increased systemic delivery
ATE209902T1 (de) 1996-08-19 2001-12-15 Us Gov Health & Human Serv Neue liposomkomplexe mit erhöhter systemischer verabreichung
TW520297B (en) 1996-10-11 2003-02-11 Sequus Pharm Inc Fusogenic liposome composition and method
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
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
ES2298269T3 (es) 2000-09-26 2008-05-16 Idera Pharmaceuticals, Inc. Modulacion de la actividad inmunoestimulante de analogos oligonucleotidicos inmunoestimulantes mediante cambios quimicos posicionales.
US7189759B2 (en) * 2001-05-23 2007-03-13 Medicis Pharmaceutical Corporation Compositions for the treatment of pigmentation disorders and methods for their manufacture
WO2003027277A1 (fr) 2001-09-21 2003-04-03 Japan Science And Technology Corporation Procede de criblage de facteur de reprogrammation, facteur de reprogrammation crible au moyen de ce procede, procede d'utilisation du facteur de reprogrammation, procede de differenciation de cellules fusionnees non differenciees et procede de construction de cellules, de tissus et d'organes
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
BRPI0410886A (pt) 2003-06-03 2006-07-04 Isis Pharmaceuticals Inc composto de filamento duplo, composição farmacêutica, sal farmaceuticamente aceitável, métodos de modificação do ácido nucleico que codifica a survivina humana, de inibição da expressão da suvivina em células ou tecidos, e de tratamento de uma condição associada com a expressão ou superexpressão da suvivina, e, oligonucleotìdeo de rnai de filamento único
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
AU2005282414C1 (en) 2004-09-08 2011-04-07 Wisconsin Alumni Research Foundation Culturing human embryonic stem cells
CN101044235B (zh) 2004-09-08 2013-01-02 威斯康星校友研究基金会 胚胎干细胞的培养基和培养
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
EP4332227A1 (en) 2005-08-23 2024-03-06 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
AU2006286149B2 (en) 2005-08-29 2012-09-13 Technion Research And Development Foundation Ltd. Media for culturing stem cells
EP2208786B1 (en) 2005-12-13 2018-08-01 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
CA2634329A1 (en) 2005-12-22 2007-07-19 Csl Behring Gmbh 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
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
CN101743306A (zh) 2007-03-23 2010-06-16 威斯康星校友研究基金会 体细胞重编程
WO2008124133A1 (en) 2007-04-07 2008-10-16 Whitehead Institute For Biomedical Research Reprogramming of somatic cells
WO2009075119A1 (ja) 2007-12-10 2009-06-18 Kyoto University 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
CA2718904C (en) 2008-03-17 2017-01-03 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
EP3447128A1 (en) 2008-06-04 2019-02-27 FUJIFILM Cellular Dynamics, 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
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
WO2010022194A2 (en) 2008-08-20 2010-02-25 Virxsys Corporation Compositions and methods for generation of pluripotent stem cells
DK3450545T3 (da) 2008-10-24 2023-10-02 Wisconsin Alumni Res Found Pluripotente stamceller opnået ved ikke-viral omprogrammering
EP2881461A1 (en) 2008-11-21 2015-06-10 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Reprogramming cells toward a pluripotent state
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
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
EP2421563B1 (en) 2009-04-22 2017-04-12 Massachusetts Institute of Technology Innate immune suppression enables repeated delivery of long rna molecules
DK2421957T3 (da) 2009-04-22 2021-01-25 Viacyte Inc Cellesammensætninger afledt af dedifferentierede omprogrammerede celler
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
ES2620960T3 (es) * 2009-06-16 2017-06-30 Curna, Inc. Tratamiento de enfermedades relacionadas con un gen de colágeno mediante la inhibición de un transcrito antisentido natural a un gen de colágeno
WO2011012316A2 (de) 2009-07-31 2011-02-03 Ludwig-Maximilians-Universität Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression
JP5898086B2 (ja) 2009-11-04 2016-04-06 セルラー ダイナミクス インターナショナル, インコーポレイテッド 化学物質を用いるエピソームリプログラミング
CA2780726A1 (en) 2009-11-11 2011-05-19 Tariq M. Rana 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
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
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
US20130145487A1 (en) 2010-05-12 2013-06-06 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
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
AU2011285531B2 (en) 2010-08-05 2015-04-30 Wisconsin Alumni Research Foundation Simplified basic media for human pluripotent cell culture
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
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
CN103502436A (zh) 2011-03-07 2014-01-08 麻省理工学院 用核酸转染细胞的方法
WO2012131090A1 (en) * 2011-03-31 2012-10-04 Galderma Research & Development Method for treatment of xeroderma pigmentosum
WO2012135805A2 (en) 2011-03-31 2012-10-04 modeRNA Therapeutics Delivery and formulation of engineered nucleic acids
WO2012138453A1 (en) 2011-04-03 2012-10-11 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
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.
RU2707251C2 (ru) 2011-10-03 2019-11-25 Модерна Терапьютикс, Инк. Модифицированные нуклеозиды, нуклеотиды и нуклеиновые кислоты и их применение
EP3279324B1 (en) * 2011-10-11 2021-02-17 Institut National De La Sante Et De La Recherche Medicale (Inserm) Exon skipping therapy for dystrophic epidermolysis bullosa
US8497124B2 (en) 2011-12-05 2013-07-30 Factor Bioscience Inc. Methods and products for reprogramming cells to a less differentiated state
RU2691027C2 (ru) * 2011-12-05 2019-06-07 Фэктор Байосайенс Инк. Способы и препараты для трансфекции клеток
KR20140102759A (ko) * 2011-12-16 2014-08-22 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드 및 핵산 조성물
WO2013096709A2 (en) 2011-12-21 2013-06-27 modeRNA Therapeutics Methods of increasing the viability or longevity of an organ or organ explant
SI3421601T1 (sl) 2011-12-30 2020-03-31 Cellscript, Llc Izdelava in uporaba in vitro-sintetizirane ssRNA za uvajanje v celice sesalca, da se inducira biološki ali biokemijski učinek
EP2833892A4 (en) 2012-04-02 2016-07-20 Moderna Therapeutics Inc MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF PROTEINS AND PEPTIDES ASSOCIATED WITH ONCOLOGY
SG11201407768QA (en) 2012-04-24 2015-01-29 Brigham & Womens Hospital Generating pluripotent cells de novo
US10155929B2 (en) 2012-05-13 2018-12-18 Allele Biotechnology & Pharmaceuticals, Inc. Feeder-free derivation of human-induced pluripotent stem cells with synthetic messenger RNA
US10119150B2 (en) 2012-05-13 2018-11-06 Allele Biotechnology & Pharmaceuticals, Inc. Feeder-free Derivation of human-induced pluripotent stem cells with synthetic messenger RNA
CN110241087B (zh) 2012-05-13 2023-02-03 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
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
MX2015007550A (es) * 2012-12-12 2017-02-02 Broad Inst Inc Suministro, modificación y optimización de sistemas, métodos y composiciones para la manipulación de secuencias y aplicaciones terapéuticas.
CA2902237C (en) 2013-02-22 2024-02-13 The Board Of Trustees Of The Leland Stanford Junior University Compounds, compositions, methods, and kits relating to telomere extension
JP6480874B2 (ja) * 2013-03-01 2019-03-13 リージェンツ・オブ・ザ・ユニバーシティ・オブ・ミネソタRegents Of The University Of Minnesota Talenに基づく遺伝子修正
US20160220613A1 (en) * 2013-09-16 2016-08-04 Agency For Science, Technology And Research Method

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