JP2012520085A - 生物活性rnaの送達のための組成物及び方法 - Google Patents

生物活性rnaの送達のための組成物及び方法 Download PDF

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JP2012520085A
JP2012520085A JP2011554275A JP2011554275A JP2012520085A JP 2012520085 A JP2012520085 A JP 2012520085A JP 2011554275 A JP2011554275 A JP 2011554275A JP 2011554275 A JP2011554275 A JP 2011554275A JP 2012520085 A JP2012520085 A JP 2012520085A
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sequence
rna
sequences
cell
polynucleotide
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JP2012520085A5 (OSRAM
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ポラック、ケヴィン
フューウェル、ジェイソン
アンワー、クワーシード
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エーゲン、インコーポレイテッド
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation

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  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
JP2011554275A 2009-03-13 2010-03-15 生物活性rnaの送達のための組成物及び方法 Pending JP2012520085A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US16028709P 2009-03-13 2009-03-13
US16028809P 2009-03-13 2009-03-13
US61/160,287 2009-03-13
US61/160,288 2009-03-13
PCT/US2010/027365 WO2010105277A1 (en) 2009-03-13 2010-03-15 Compositions and methods for the delivery of biologically active rnas

Publications (2)

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JP2012520085A true JP2012520085A (ja) 2012-09-06
JP2012520085A5 JP2012520085A5 (OSRAM) 2013-02-21

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US (2) US8569065B2 (OSRAM)
EP (1) EP2406379A4 (OSRAM)
JP (1) JP2012520085A (OSRAM)
KR (1) KR20110128345A (OSRAM)
CN (1) CN102625842A (OSRAM)
AU (1) AU2010223888A1 (OSRAM)
CA (1) CA2755245A1 (OSRAM)
WO (1) WO2010105277A1 (OSRAM)

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US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
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EP4144378A1 (en) 2011-12-16 2023-03-08 ModernaTX, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
CN104169419A (zh) * 2011-12-23 2014-11-26 艾根股份有限公司 用于输送生物活性rna的组合物和方法
US20140371302A1 (en) * 2011-12-29 2014-12-18 Modema Therapeutics, Inc. Modified mrnas encoding cell-penetrating polypeptides
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US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
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US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
WO2014144423A2 (en) * 2013-03-15 2014-09-18 Techulon Inc. Antisense molecules for treatment of staphylococcus aureus infection
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KR20160042917A (ko) 2013-07-25 2016-04-20 엑시큐어, 인크. 예방 및 치료 용도를 위한 면역자극제로서의 구형 핵산-기재 구축물
US10568898B2 (en) 2013-08-13 2020-02-25 Northwestern University Lipophilic nanoparticles for drug delivery
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
BR112016007255A2 (pt) 2013-10-03 2017-09-12 Moderna Therapeutics Inc polinucleotídeos que codificam receptor de lipoproteína de baixa densidade
EP3184641B1 (en) * 2013-10-28 2020-07-08 University of Pittsburgh - Of the Commonwealth System of Higher Education Oncolytic hsv vector
US10434064B2 (en) 2014-06-04 2019-10-08 Exicure, Inc. Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications
CN106794256B (zh) 2014-08-19 2021-04-30 西北大学 蛋白质/寡核苷酸核-壳纳米颗粒治疗剂
WO2016057549A1 (en) 2014-10-06 2016-04-14 Aurasense Therapeutics, Llc Anti-tnf compounds
US9816080B2 (en) * 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
US10078092B2 (en) 2015-03-18 2018-09-18 Northwestern University Assays for measuring binding kinetics and binding capacity of acceptors for lipophilic or amphiphilic molecules
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US11639371B2 (en) * 2016-02-17 2023-05-02 The Chinese University Of Hong Kong Peptidylic inhibitors of nucleolin (NCL) targeting CAG-repeat RNA toxicity and methods for reducing polyQ-mediated toxicity in polyQ diseases
JP2019515654A (ja) 2016-03-16 2019-06-13 ザ ジェイ. デヴィッド グラッドストーン インスティテューツ 肥満及び/又は糖尿病を処置するための方法及び組成物、並びに候補処置薬剤を識別するための方法及び組成物
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WO2020010083A1 (en) * 2018-07-05 2020-01-09 The Regents Of The University Of California Compositions and methods for delivery of rna to a cell
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WO2023244760A1 (en) * 2022-06-15 2023-12-21 Duke University Protein library display systems and methods thereof

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Publication number Priority date Publication date Assignee Title
JP2017505638A (ja) * 2014-02-14 2017-02-23 シンヴィヴォ コーポレイション ハイブリッドタンパクおよびその使用

Also Published As

Publication number Publication date
CN102625842A (zh) 2012-08-01
WO2010105277A1 (en) 2010-09-16
US20100233141A1 (en) 2010-09-16
CA2755245A1 (en) 2010-09-16
KR20110128345A (ko) 2011-11-29
US20140005254A1 (en) 2014-01-02
EP2406379A1 (en) 2012-01-18
AU2010223888A1 (en) 2011-10-06
US8569065B2 (en) 2013-10-29
EP2406379A4 (en) 2012-09-26

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