BR112016017736A2 - métodos e produtos para produção e distribuição de ácido nucleico - Google Patents

métodos e produtos para produção e distribuição de ácido nucleico

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Publication number
BR112016017736A2
BR112016017736A2 BR112016017736A BR112016017736A BR112016017736A2 BR 112016017736 A2 BR112016017736 A2 BR 112016017736A2 BR 112016017736 A BR112016017736 A BR 112016017736A BR 112016017736 A BR112016017736 A BR 112016017736A BR 112016017736 A2 BR112016017736 A2 BR 112016017736A2
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BR
Brazil
Prior art keywords
methods
nucleic acids
cells
products
therapeutic
Prior art date
Application number
BR112016017736A
Other languages
English (en)
Inventor
Rohde Christopher
Angel Matthew
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 BR112016017736A2 publication Critical patent/BR112016017736A2/pt

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    • A61K31/573Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
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    • A61K31/575Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of three or more carbon atoms, e.g. cholane, cholestane, ergosterol, sitosterol
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    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor

Abstract

a presente invenção refere-se, em parte, aos ácidos nucleicos, incluindo ácidos nucleicos que codificam proteínas, agentes terapêuticos e cosméticos que compreendem ácidos nucleicos, métodos para distribuição de ácidos nucleicos para células, tecidos, órgãos e pacientes, métodos para induzir as células para que expressem proteínas utilizando ácidos nucleicos, métodos, kits e dispositivos para transfecção, edição gênica e reprogramação de células, e células, organismos, agentes terapêuticos e cosméticos produzidos utilizando estes métodos, kits e dispositivos. são descritos métodos e produtos para alterar a sequência de dna de uma célula, assim como os métodos e produtos para induzir as células a expressarem proteínas usando moléculas de rna sintéticos, incluindo células presentes in vivo. também são descritos produtos terapêuticos compreendendo ácidos nucleicos que codificam proteínas de edição gênica.
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Families Citing this family (60)

* 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
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
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3212793B1 (en) 2014-11-02 2020-01-08 Arcturus Therapeutics, Inc. Messenger una molecules and uses thereof
EP3256585A4 (en) * 2015-02-13 2018-08-15 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
IL258821B (en) 2015-10-23 2022-07-01 Harvard College Nucleobase editors and their uses
CA3011692A1 (en) 2015-11-23 2017-06-01 The Regents Of The University Of Colorado, A Body Corporate Methods and compositions for reprogramming cells
DK3377637T3 (da) 2016-04-08 2020-05-18 Krystal Biotech Inc Sammensætninger til anvendelse i fremgangsmåder til behandling af sår, lidelser og sygdomme i huden
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
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
US10576167B2 (en) 2016-08-17 2020-03-03 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
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
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
CN110582304A (zh) * 2016-12-08 2019-12-17 库尔维科公司 用于治疗或预防肝脏疾病的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
WO2018165504A1 (en) 2017-03-09 2018-09-13 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
GB2575930A (en) 2017-03-23 2020-01-29 Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
AU2018246292A1 (en) 2017-03-31 2019-10-10 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
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
CA3067382A1 (en) 2017-06-15 2018-12-20 The Regents Of The University Of California Targeted non-viral dna insertions
JP2020534795A (ja) 2017-07-28 2020-12-03 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 ファージによって支援される連続的進化(pace)を用いて塩基編集因子を進化させるための方法および組成物
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
EP3676376A2 (en) 2017-08-30 2020-07-08 President and Fellows of Harvard College High efficiency base editors comprising gam
CN111278447A (zh) * 2017-09-05 2020-06-12 阿兹特拉公司 用重组微生物治疗炎性皮肤疾病的方法和组合物
CA3078631A1 (en) * 2017-10-11 2019-04-18 Jacob M. Waugh Methods and compositions for topical delivery
WO2019079347A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. USES OF BASIC EDITORS ADENOSINE
MX2020004325A (es) 2017-10-27 2020-11-09 Univ California Reemplazo dirigido de receptores de células t endógenos.
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
CN112236153A (zh) 2018-04-05 2021-01-15 阿兹特拉公司 用重组微生物治疗皮肤疾病的方法和组合物
CA3094345A1 (en) 2018-04-12 2019-10-17 Krystal Biotech, Inc. Compositions and methods for the treatment of autosomal recessive congenital ichthyosis
US10786438B2 (en) * 2018-04-27 2020-09-29 Krystal Biotech, Inc. Recombinant nucleic acids encoding cosmetic protein(s) for aesthetic applications
CN112867792A (zh) 2018-08-23 2021-05-28 桑格摩生物治疗股份有限公司 工程化靶特异性碱基编辑器
CA3112627A1 (en) 2018-09-24 2020-04-02 Krystal Biotech, Inc. Compositions and methods for the treatment of netherton syndrome
US20210395775A1 (en) * 2018-09-26 2021-12-23 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
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
WO2020191249A1 (en) 2019-03-19 2020-09-24 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
CA3231676A1 (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
US20230285546A1 (en) * 2022-01-05 2023-09-14 Helix Nanotechnologies, Inc. Compositions comprising alpha-factor prepro sequence and uses thereof
CN114480365A (zh) * 2022-01-18 2022-05-13 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
ATE209902T1 (de) 1996-08-19 2001-12-15 Us Gov Health & Human Serv Neue liposomkomplexe mit erhöhter systemischer verabreichung
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
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
JP2002508299A (ja) 1997-09-19 2002-03-19 セクイター, インク. センスmRNA治療
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
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
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
AU2002330465B2 (en) 2001-09-21 2006-07-27 Kyoto University Screening method for a reprogramming agent, reprogramming agent screened by the method, method for using the reprogramming agent, method for differentiating an undifferentiated fusion cell, and production method of cells, 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
CN1984921B (zh) 2003-06-03 2010-06-16 Isis药物公司 存活蛋白表达的调节
CA2546616A1 (en) 2003-11-21 2005-06-09 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
NZ553241A (en) 2004-09-08 2009-11-27 Wisconsin Alumni Res Found Medium and culture of pluripotent stem cells
AU2005282414C1 (en) 2004-09-08 2011-04-07 Wisconsin Alumni Research Foundation Culturing human embryonic stem cells
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
ES2937245T3 (es) 2005-08-23 2023-03-27 Univ Pennsylvania ARN que contiene nucleósidos modificados y métodos de uso del mismo
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
EP3354723B1 (en) 2005-08-29 2023-12-13 Technion Research & Development Foundation Ltd. Media for culturing stem cells
EP4223769A3 (en) 2005-12-13 2023-11-01 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
US8278104B2 (en) 2005-12-13 2012-10-02 Kyoto University Induced pluripotent stem cells produced with Oct3/4, Klf4 and Sox2
EP1966239A1 (de) 2005-12-22 2008-09-10 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
AU2007333225B2 (en) 2006-12-08 2014-06-12 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
US8859229B2 (en) 2007-02-02 2014-10-14 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
US8440461B2 (en) 2007-03-23 2013-05-14 Wisconsin Alumni Research Foundation Reprogramming somatic cells using retroviral vectors comprising Oct-4 and Sox2 genes
WO2008124133A1 (en) 2007-04-07 2008-10-16 Whitehead Institute For Biomedical Research Reprogramming of somatic cells
AU2008286249B2 (en) 2007-12-10 2013-10-10 Kyoto University Efficient method for nuclear reprogramming
EP2072618A1 (en) 2007-12-14 2009-06-24 Johannes Gutenberg-Universität Mainz Use of RNA for reprogramming somatic cells
SG10202103401QA (en) 2008-03-17 2021-05-28 Scripps Research Inst 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
AU2009256202B2 (en) 2008-06-04 2014-07-03 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
US20110263015A1 (en) 2008-08-20 2011-10-27 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
ES2561949T3 (es) 2008-11-21 2016-03-01 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Reprogramación de células hacia un estado pluripotente
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
EP2421563B1 (en) 2009-04-22 2017-04-12 Massachusetts Institute of Technology Innate immune suppression enables repeated delivery of long rna molecules
US10837020B2 (en) 2009-04-22 2020-11-17 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long RNA molecules
US20100272695A1 (en) 2009-04-22 2010-10-28 Alan Agulnick Cell compositions derived from dedifferentiated reprogrammed cells
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
KR101801404B1 (ko) * 2009-06-16 2017-12-20 큐알엔에이, 인크. 콜라겐 유전자에 대한 천연 안티센스 전사체의 억제에 의한 콜라겐 유전자 관련된 질환의 치료
WO2011012316A2 (de) 2009-07-31 2011-02-03 Ludwig-Maximilians-Universität Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
US9295697B2 (en) 2009-11-04 2016-03-29 Cellular Dynamics International, 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
DK3112467T3 (en) 2009-12-07 2018-04-23 Univ Pennsylvania RNA PREPARATIONS COMPREHENSIVE PURIFIED MODIFIED RNA FOR CELL PROGRAMMING
US20130189741A1 (en) 2009-12-07 2013-07-25 Cellscript, Inc. Compositions and methods for reprogramming mammalian cells
SG181601A1 (en) 2009-12-10 2012-07-30 Univ 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
US8048675B1 (en) 2010-05-12 2011-11-01 Ipierian, Inc. Integration-free human induced pluripotent stem cells from blood
WO2011141820A1 (en) 2010-05-12 2011-11-17 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene 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
EP2601288B1 (en) 2010-08-05 2016-04-06 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
EP2616540A4 (en) 2010-09-14 2014-02-19 Univ Kyoto METHOD OF EFFICIENTLY ESTABLISHING INDUCED PLURIPOTENT STEM CELLS
DK3590949T3 (da) 2010-10-01 2022-07-11 Modernatx Inc Ribonukleinsyrer indeholdende n1-methyl-pseudouracil og anvendelse heraf
WO2012048213A1 (en) 2010-10-08 2012-04-12 Regents Of The University Of Minnesota A method to increase gene targeting frequency
US9637732B2 (en) 2010-11-04 2017-05-02 Kyoto University Method of efficiently establishing induced pluripotent stem cells
CA2832807A1 (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
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
EP2694660B1 (en) 2011-04-03 2018-08-08 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
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
JP2014524241A (ja) 2011-08-03 2014-09-22 ロータス ティシュー リペア インコーポレイテッド コラーゲン7及び関連する方法
SG10201602654SA (en) 2011-10-03 2016-05-30 Moderna Therapeutics Inc Modified nucleosides,nucleotides,and nucleic acids,and uses thereof
ES2867054T3 (es) * 2011-10-11 2021-10-20 Inst Nat Sante Rech Med Tratamiento de salto de exones para epidermólisis ampollosa distrófica
US8497124B2 (en) 2011-12-05 2013-07-30 Factor Bioscience Inc. Methods and products for reprogramming cells to a less differentiated state
AU2012347919B2 (en) * 2011-12-05 2017-02-02 Factor Bioscience Inc. Methods and products for transfecting cells
WO2013090648A1 (en) 2011-12-16 2013-06-20 modeRNA Therapeutics Modified nucleoside, nucleotide, and nucleic acid compositions
CA2859691A1 (en) 2011-12-21 2013-06-27 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
JP2015516143A (ja) 2012-04-02 2015-06-08 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. ヒト疾患に関連するタンパク質の産生のための修飾ポリヌクレオチド
JP2015516812A (ja) 2012-04-24 2015-06-18 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッドThe Brigham and Women’s Hospital, Inc. 多能性細胞のデノボ生成
JP6448531B2 (ja) 2012-05-13 2019-01-09 アリール バイオテクノロジー アンド ファーマシューティカルズ, インコーポレイテッド 合成メッセンジャーrnaを使用したヒト人工多能性幹細胞のフィーダーフリー誘導
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
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
SG10201912328UA (en) * 2012-12-12 2020-02-27 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
RU2766120C2 (ru) 2013-02-22 2022-02-08 Дзе Борд Оф Трастиз Оф Дзе Лелэнд Стэнфорд Джуниор Юниверсити Соединения, композиции, способы и наборы, связанные с удлинением теломер
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
SG11201601939VA (en) * 2013-09-16 2016-04-28 Agency Science Tech & Res Method

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