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WO2012018881A3 - Methods and compositions for the regulation of rna - Google Patents

Methods and compositions for the regulation of rna Download PDF

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Publication number
WO2012018881A3
WO2012018881A3 PCT/US2011/046365 US2011046365W WO2012018881A3 WO 2012018881 A3 WO2012018881 A3 WO 2012018881A3 US 2011046365 W US2011046365 W US 2011046365W WO 2012018881 A3 WO2012018881 A3 WO 2012018881A3
Authority
WO
WIPO (PCT)
Prior art keywords
compositions
methods
rna
regulation
lncrna
Prior art date
Legal status (The legal status 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 status listed.)
Ceased
Application number
PCT/US2011/046365
Other languages
French (fr)
Other versions
WO2012018881A2 (en
Inventor
Kenneth S. Koblan
Stuart Pollard
Gregory Hinkle
Brian Bettencourt
Donna T. Ward
Muthiah Manoharan
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Alnylam Pharmaceuticals Inc
Original Assignee
Alnylam Pharmaceuticals 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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of WO2012018881A2 publication Critical patent/WO2012018881A2/en
Publication of WO2012018881A3 publication Critical patent/WO2012018881A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/11DNA 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The invention relates to compositions and methods of modulating the status, activity or expression of long intervening non-coding RNA transcript (lncRNA) targets.
PCT/US2011/046365 2010-08-03 2011-08-03 Methods and compositions for the regulation of rna Ceased WO2012018881A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US37041410P 2010-08-03 2010-08-03
US61/370,414 2010-08-03

Publications (2)

Publication Number Publication Date
WO2012018881A2 WO2012018881A2 (en) 2012-02-09
WO2012018881A3 true WO2012018881A3 (en) 2012-08-09

Family

ID=45560043

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2011/046365 Ceased WO2012018881A2 (en) 2010-08-03 2011-08-03 Methods and compositions for the regulation of rna

Country Status (1)

Country Link
WO (1) WO2012018881A2 (en)

Cited By (4)

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US9175291B2 (en) 2012-10-11 2015-11-03 Isis Pharmaceuticals Inc. Modulation of androgen receptor expression
US9328346B2 (en) 2010-11-12 2016-05-03 The General Hospital Corporation Polycomb-associated non-coding RNAs
CN109001188B (en) * 2018-08-12 2021-09-14 河北农业大学 Specific molecularly imprinted polymer of adamantanamine and rimantadine, chemiluminescence kit, detection method and application
US12139617B2 (en) 2017-12-22 2024-11-12 North Carolina State University Polymeric fluorophores, compositions comprising the same, and methods of preparing and using the same

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US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
EP2831231A1 (en) 2012-03-30 2015-02-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
US10174315B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating hemoglobin gene family expression
AU2013262649A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating smn gene family expression
AU2013262663A1 (en) * 2012-05-16 2015-01-22 The General Hospital Corporation D/B/A Massachusetts General Hospital Compositions and methods for modulating gene expression
EP2850190B1 (en) 2012-05-16 2020-07-08 Translate Bio MA, Inc. Compositions and methods for modulating mecp2 expression
EP2850185A4 (en) 2012-05-16 2015-12-30 Rana Therapeutics Inc COMPOSITIONS AND METHODS FOR MODULATING UTRN EXPRESSION
CA2873766A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating atp2a2 expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
US9988625B2 (en) 2013-01-10 2018-06-05 Dharmacon, Inc. Templates, libraries, kits and methods for generating molecules
EP3766974A1 (en) 2013-03-14 2021-01-20 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating tau expression
EP2813578A1 (en) * 2013-06-14 2014-12-17 Prestizia Methods for detecting an infectious agent, in particular HIV1, using long noncoding RNA
TW202246503A (en) 2013-07-19 2022-12-01 美商百健Ma公司 Compositions for modulating tau expression
CA2917714A1 (en) * 2013-08-07 2015-02-12 Regeneron Pharmaceuticals, Inc. Lincrna-deficient non-human animals
NO3036326T3 (en) * 2013-08-20 2018-03-03
CA2923029A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
EA201690675A1 (en) 2013-10-03 2016-08-31 Модерна Терапьютикс, Инк. POLYNUCLEOTES ENCODING THE RECEPTOR OF LOW DENSITY LIPOPROTEINS
EP3169693B1 (en) 2014-07-16 2022-03-09 ModernaTX, Inc. Chimeric polynucleotides
EP3171895A1 (en) 2014-07-23 2017-05-31 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
CA2966044A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
CN112410339A (en) 2014-11-14 2021-02-26 沃雅戈治疗公司 regulatory polynucleotide
EP3245221A4 (en) 2015-01-16 2018-06-13 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
US10900036B2 (en) 2015-03-17 2021-01-26 The General Hospital Corporation RNA interactome of polycomb repressive complex 1 (PRC1)
WO2016172734A1 (en) * 2015-04-24 2016-10-27 California Institute Of Technology Reactivation of x chromosome genes
CA3002744A1 (en) * 2015-10-19 2017-04-27 Rxi Pharmaceuticals Corporation Reduced size self-delivering nucleic acid compounds targeting long non-coding rna
EP3368054A4 (en) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. REGULATORY EXPRESSION USING AN ADENO-ASSOCIATED VIRUS (AAV)
CN105567806B (en) * 2015-12-07 2018-11-20 中山大学附属第一医院 Application of NR _027669.1 in detection of fungal infection of organ transplant patient and kit
CN105567804B (en) * 2015-12-07 2018-11-20 中山大学附属第一医院 Application of NR _073415.2 in detecting cytomegalovirus infection of organ transplantation patient and kit
JOP20190065A1 (en) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc Compounds and Methods for Reducing the Expression of TAU
EP3532044A4 (en) 2016-10-27 2020-07-29 California Institute of Technology HDAC-INHIBITING COMPOSITIONS FOR REACTIVATING THE X-CHROMOSOME
WO2018098352A2 (en) 2016-11-22 2018-05-31 Jun Oishi Targeting kras induced immune checkpoint expression
US20200131498A1 (en) 2017-06-14 2020-04-30 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase
KR102477906B1 (en) * 2017-11-29 2022-12-15 연세대학교 산학협력단 A lncRNA FOR INDUCED PLURIPOTENT STEM CELL DIFFERENTIATION AND USES THEREOF
JP2021505175A (en) * 2017-12-11 2021-02-18 ロシュ イノベーション センター コペンハーゲン エーエス Oligonucleotides for regulating the expression of FNDC3B
WO2019126651A1 (en) * 2017-12-21 2019-06-27 Alnylam Pharmaceuticals, Inc. Chirally-enriched double-stranded rna agents
US20210010028A1 (en) 2018-03-06 2021-01-14 Voyager Therapeutics, Inc. Insect cell manufactured partial self-complementary aav genomes
EP3778892A4 (en) * 2018-04-10 2022-01-19 Ractigen Therapeutics Novel small activating rna
SG11202108450SA (en) 2019-02-27 2021-09-29 Ionis Pharmaceuticals Inc Modulators of malat1 expression
GB201907380D0 (en) * 2019-05-24 2019-07-10 Cancer Research Tech Ltd Mediators of gene silencing
US20230190785A1 (en) * 2020-03-30 2023-06-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
WO2021224475A1 (en) * 2020-05-07 2021-11-11 Xandrax Ab Cancer treatment and diagnosis involving lincrna
CN113201590B (en) * 2020-08-05 2024-05-07 上海健康医学院 LncRNA for evaluating early recurrence risk of hepatocellular carcinoma, evaluation method and device
IT202000023647A1 (en) * 2020-10-07 2022-04-07 Univ Degli Studi Padova SIRNA IN THE TREATMENT OF ECTRODACTYLY-ECTODERMAL DYSPLASIA-CLEANING SYNDROME (EEC)
EP4573198A2 (en) * 2022-08-18 2025-06-25 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
CN117180297B (en) * 2023-09-08 2024-09-20 广州医科大学附属第一医院(广州呼吸中心) Markers for diagnosis, treatment and prognosis assessment of prostate cancer and their applications

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US20050153337A1 (en) * 2003-04-03 2005-07-14 Muthiah Manoharan iRNA conjugates
US20060240093A1 (en) * 2003-07-16 2006-10-26 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
US20060134663A1 (en) * 2004-11-03 2006-06-22 Paul Harkin Transcriptome microarray technology and methods of using the same

Non-Patent Citations (3)

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FORREST ET AL.: "Annotating non-coding transcription using functional genomics strategies.", BRIEF FUNCT. GENOMIC PROTEOMIC, vol. 8, no. 6, 2009, pages 437 - 443 *
GUPTA ET AL.: "Long non-coding RNA HOTAIR reprograms chromatin state to promote cancer metastasis.", NATURE, vol. 464, no. 7291, 15 April 2010 (2010-04-15), pages 1071 - 1076, XP055037476, DOI: doi:10.1038/nature08975 *
KHALIL ET AL.: "Many human large intergenic noncoding RNAs associate with chromatin- modifying complexes and affect gene expression.", PROC. NATL. ACAD. SCI., vol. 106, no. 28, 2009, pages 11667 - 11672, XP055073351, DOI: doi:10.1073/pnas.0904715106 *

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9328346B2 (en) 2010-11-12 2016-05-03 The General Hospital Corporation Polycomb-associated non-coding RNAs
US9175291B2 (en) 2012-10-11 2015-11-03 Isis Pharmaceuticals Inc. Modulation of androgen receptor expression
US12139617B2 (en) 2017-12-22 2024-11-12 North Carolina State University Polymeric fluorophores, compositions comprising the same, and methods of preparing and using the same
CN109001188B (en) * 2018-08-12 2021-09-14 河北农业大学 Specific molecularly imprinted polymer of adamantanamine and rimantadine, chemiluminescence kit, detection method and application

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Publication number Publication date
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