CN102625842A - 用于输送生物活性rna的组合物和方法 - Google Patents

用于输送生物活性rna的组合物和方法 Download PDF

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Publication number
CN102625842A
CN102625842A CN2010800210226A CN201080021022A CN102625842A CN 102625842 A CN102625842 A CN 102625842A CN 2010800210226 A CN2010800210226 A CN 2010800210226A CN 201080021022 A CN201080021022 A CN 201080021022A CN 102625842 A CN102625842 A CN 102625842A
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sequence
rna
cell
biologically active
polynucleotide
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CN2010800210226A
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Chinese (zh)
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K.波拉科
J.费维尔
K.安沃
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CLSN LAB Inc
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Egen Inc
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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
    • 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
    • C12N15/11DNA 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
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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)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
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  • 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)
CN2010800210226A 2009-03-13 2010-03-15 用于输送生物活性rna的组合物和方法 Pending CN102625842A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US16028809P 2009-03-13 2009-03-13
US16028709P 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 (1)

Publication Number Publication Date
CN102625842A true CN102625842A (zh) 2012-08-01

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Application Number Title Priority Date Filing Date
CN2010800210226A Pending CN102625842A (zh) 2009-03-13 2010-03-15 用于输送生物活性rna的组合物和方法

Country Status (8)

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

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US9880151B2 (en) * 2011-05-23 2018-01-30 Phylogica Limited Method of determining, identifying or isolating cell-penetrating peptides
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2763701B1 (en) 2011-10-03 2018-12-19 Moderna Therapeutics, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
KR20140102759A (ko) 2011-12-16 2014-08-22 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드 및 핵산 조성물
WO2013096958A1 (en) * 2011-12-23 2013-06-27 Egen, Inc. Compositions and methods for the delivery of biologically active rnas
WO2013101690A1 (en) * 2011-12-29 2013-07-04 modeRNA Therapeutics Modified mrnas encoding cell-penetrating polypeptides
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9878056B2 (en) 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
DE18200782T1 (de) 2012-04-02 2021-10-21 Modernatx, Inc. Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
CA2892529C (en) 2012-11-26 2023-04-25 Moderna Therapeutics, Inc. Terminally modified rna
KR20160021076A (ko) * 2013-03-15 2016-02-24 테출론 인코포레이티드 스타필로코커스 아우레우스 감염의 치료를 위한 안티센스 분자
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
WO2015002956A1 (en) * 2013-07-01 2015-01-08 Ohio State Innovation Foundation Exosome delivery system
WO2015013673A1 (en) 2013-07-25 2015-01-29 Aurasense Therapeutics, Llc Spherical nucleic acid-based constructs as immunostimulatory agents for prophylactic and therapeutic use
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
EA201690675A1 (ru) 2013-10-03 2016-08-31 Модерна Терапьютикс, Инк. Полинуклеотиды, кодирующие рецептор липопротеинов низкой плотности
JP6588024B2 (ja) 2013-10-28 2019-10-09 ユニヴァーシティ オヴ ピッツバーグ オヴ ザ コモンウェルス システム オヴ ハイアー エデュケーション 腫瘍溶解性hsvベクター
JP6744224B2 (ja) 2014-02-14 2020-08-19 シンヴィヴォ コーポレイション 核酸ベクターおよびその使用
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
EP3183007B1 (en) 2014-08-19 2020-06-17 Northwestern University Protein/oligonucleotide core-shell nanoparticle therapeutics
JP6741673B2 (ja) 2014-10-06 2020-08-19 イグジキュア, インコーポレーテッドExicure, Inc. 抗tnf化合物
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
CN109414414A (zh) 2016-03-16 2019-03-01 戴维·格拉德斯通研究所 用于治疗肥胖症和/或糖尿病以及用于鉴定候选治疗剂的方法和组合物
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US20050260756A1 (en) * 2003-01-28 2005-11-24 Troy Carol M Complex for facilitating delivery of dsRNA into a cell and uses thereof
CN1940074A (zh) * 2006-08-07 2007-04-04 汪运山 胃癌靶向AFP基因的siRNAs表达载体的构建、筛选及其用途
WO2007126386A1 (en) * 2006-05-03 2007-11-08 Loetvall Jan Olof Exosome transfer of nucleic acids to cells

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Also Published As

Publication number Publication date
US20100233141A1 (en) 2010-09-16
WO2010105277A1 (en) 2010-09-16
AU2010223888A1 (en) 2011-10-06
CA2755245A1 (en) 2010-09-16
US20140005254A1 (en) 2014-01-02
JP2012520085A (ja) 2012-09-06
US8569065B2 (en) 2013-10-29
EP2406379A4 (en) 2012-09-26
EP2406379A1 (en) 2012-01-18
KR20110128345A (ko) 2011-11-29

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