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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China
Prior art keywords
sequence
rna
cell
biologically active
polynucleotide
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CN2010800210226A
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English (en)
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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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction 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)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • 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)
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
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 (1)

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

Family

ID=42728852

Family Applications (1)

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)

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

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US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
DE19216461T1 (de) 2011-10-03 2021-10-07 Modernatx, Inc. Modifizierte nukleoside, nukleotide und nukleinsäuren und verwendungen davon
CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
AU2012358181A1 (en) * 2011-12-23 2014-07-10 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
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
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US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
PL2922554T3 (pl) 2012-11-26 2022-06-20 Modernatx, Inc. Na zmodyfikowany na końcach
US9909123B2 (en) * 2013-03-15 2018-03-06 Techulon Inc. Antisense molecules for treatment of Staphylococcus aureus infection
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
EP3024936B1 (en) 2013-07-25 2019-09-04 Exicure, Inc. 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
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052521A1 (en) 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
BR112016009465A8 (pt) 2013-10-28 2018-01-30 Univ Pittsburgh Commonwealth Sys Higher Education vetor de hsv oncolítico.
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
TR201908550T4 (tr) 2014-06-04 2019-07-22 Exicure Inc Profilaktik veya terapötik uygulamalar için lipozomal sferik nükleik asitler tarafından immün modülatörlerin çok değerlikli teslimi.
WO2016028940A1 (en) 2014-08-19 2016-02-25 Northwestern University Protein/oligonucleotide core-shell nanoparticle therapeutics
CR20170181A (es) 2014-10-06 2017-05-31 Exicure Inc Compuestos anti-tnf
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
KR20180136435A (ko) 2016-01-27 2018-12-24 온코루스, 인크. 종양 살상형 바이러스 벡터 및 그의 용도
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
JP2012520085A (ja) 2012-09-06
US20140005254A1 (en) 2014-01-02
AU2010223888A1 (en) 2011-10-06
EP2406379A4 (en) 2012-09-26
EP2406379A1 (en) 2012-01-18
CA2755245A1 (en) 2010-09-16
KR20110128345A (ko) 2011-11-29
US8569065B2 (en) 2013-10-29
WO2010105277A1 (en) 2010-09-16
US20100233141A1 (en) 2010-09-16

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ASS Succession or assignment of patent right

Owner name: CLSN LABORATORIES, INC.

Free format text: FORMER OWNER: EGEN INC.

Effective date: 20150203

C41 Transfer of patent application or patent right or utility model
TA01 Transfer of patent application right

Effective date of registration: 20150203

Address after: Delaware

Applicant after: CLSN LAB INC

Address before: alabama

Applicant before: Expression Genetics Inc.

C02 Deemed withdrawal of patent application after publication (patent law 2001)
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20120801