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

Methods and compositions for the regulation of rna Download PDF

Info

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
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
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

Links

Classifications

    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; 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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (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 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 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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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) 2018-12-18 2024-11-12 North Carolina State University Polymeric fluorophores, compositions comprising the same, and methods of preparing and using the same

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
CA2866392C (en) 2012-03-30 2023-10-24 Washington University Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
EA201492121A1 (en) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. COMPOSITIONS AND METHODS FOR MODULATING THE EXPRESSION OF THE FAMILY OF HEMOGLOBIN GENES
KR20150030205A (en) 2012-05-16 2015-03-19 라나 테라퓨틱스, 인크. Compositions and methods for modulating smn gene family expression
EP2850189B8 (en) * 2012-05-16 2019-01-23 Translate Bio MA, Inc. Compositions and methods for modulating gene expression
WO2013173645A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating utrn expression
BR112014028644A2 (en) 2012-05-16 2017-08-15 Rana Therapeutics Inc COMPOSITIONS AND METHODS FOR MODULATION OF ATP2A2 EXPRESSION
EP2850190B1 (en) 2012-05-16 2020-07-08 Translate Bio MA, Inc. Compositions and methods for modulating mecp2 expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
LT2943579T (en) * 2013-01-10 2018-11-12 Dharmacon, Inc. Libraries and methods for generating molecules
WO2014153236A1 (en) 2013-03-14 2014-09-25 Isis 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
BR112016002385A2 (en) 2013-08-07 2017-09-12 Harvard College nonhuman animal, cell, tissue or embryo, targeting vector, targeting vector construct, and methods for producing a nonhuman animal and for modifying an incrna locus in a pluripotent cell
WO2015024986A1 (en) * 2013-08-20 2015-02-26 Vib Vzw INHIBITION OF A lncRNA FOR TREATMENT OF MELANOMA
EP3041934A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2015051214A1 (en) 2013-10-03 2015-04-09 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
WO2016011226A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2016014846A1 (en) 2014-07-23 2016-01-28 Moderna Therapeutics, Inc. Modified polynucleotides for the production of intrabodies
US10858650B2 (en) 2014-10-30 2020-12-08 The General Hospital Corporation Methods for modulating ATRX-dependent gene repression
RU2719192C2 (en) 2014-11-14 2020-04-17 Вояджер Терапьютикс, Инк. Modulating polynucleotides
EP3632923A1 (en) 2015-01-16 2020-04-08 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
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
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
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
JOP20190065A1 (en) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc Compounds and methods for reducing tau expression
US11197881B2 (en) 2016-10-27 2021-12-14 California Institute Of Technology HDAC inhibitor compositions for reactivation of the X chromosome
WO2018098352A2 (en) 2016-11-22 2018-05-31 Jun Oishi Targeting kras induced immune checkpoint expression
WO2018231990A2 (en) 2017-06-14 2018-12-20 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
EP3724333A2 (en) * 2017-12-11 2020-10-21 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating fndc3b expression
WO2019126651A1 (en) * 2017-12-21 2019-06-27 Alnylam Pharmaceuticals, Inc. Chirally-enriched double-stranded rna agents
WO2019173434A1 (en) 2018-03-06 2019-09-12 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)
WO2024039776A2 (en) * 2022-08-18 2024-02-22 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） Marker for diagnosis, treatment and prognosis evaluation of prostate cancer and application thereof

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050064454A1 (en) * 2000-06-05 2005-03-24 Young Paul E. Cancer gene determination and therapeutic screening using signature gene sets
US20050153337A1 (en) * 2003-04-03 2005-07-14 Muthiah Manoharan iRNA conjugates
US20060134663A1 (en) * 2004-11-03 2006-06-22 Paul Harkin Transcriptome microarray technology and methods of using the same
US20060240093A1 (en) * 2003-07-16 2006-10-26 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050064454A1 (en) * 2000-06-05 2005-03-24 Young Paul E. Cancer gene determination and therapeutic screening using signature gene sets
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)

* Cited by examiner, † Cited by third party
Title
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
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) 2018-12-18 2024-11-12 North Carolina State University Polymeric fluorophores, compositions comprising the same, and methods of preparing and using the same

Also Published As

Publication number Publication date
WO2012018881A2 (en) 2012-02-09

Similar Documents

Publication Publication Date Title
WO2012018881A3 (en) Methods and compositions for the regulation of rna
WO2014028429A3 (en) Enzymes and polymerases for the synthesis of rna
WO2012087288A3 (en) Metal salt compositions
WO2012125544A3 (en) Necroptosis inhibitors and methods of use therefor
CA2792561C (en) Compositions and methods for inhibiting expression of cd274/pd-l1 gene
WO2012056457A3 (en) Compositions and methods for activating expression by a specific endogenous mirna
WO2011103584A3 (en) Novel ctla4-ig immunoadhesins
WO2011017108A3 (en) Cyclopropyl modulators of p2y12 receptor
WO2013155204A3 (en) Compositions and methods for inhibiting expression of the alas1 gene
WO2012068380A3 (en) Methods and compositions for modulating pd1
MX2018013216A (en) Compositions and methods for inhibiting expression of tmprss6 gene.
WO2008152131A3 (en) Rnai inhibition of alpha-enac expression
WO2010120757A3 (en) Regeneration of tissue without cell transplantation
WO2011133504A3 (en) Nanozymes, methods of making nanozymes, and methods of using nanozymes
WO2013023084A3 (en) Methods and compositions for inhibiting the growth and/or proliferation of myc-driven tumor cells
WO2011160052A3 (en) Methods and compositions for sequence specific rna endonucleases
WO2010147830A3 (en) Aminothiazole modulators of beta-3-adrenoreceptor
WO2010118291A3 (en) Biphenyl-3-carboxylic acid modulators of beta-3-adrenoreceptor
WO2011126842A3 (en) Targeting micrornas for the treatment of cardiac disorders
WO2012051117A3 (en) Substituted benzamides and their uses
WO2012017321A3 (en) Treatment for dyslipidemia
WO2012104240A3 (en) Cosmetic use
WO2013053857A3 (en) Gene cluster for biosynthesis of griselimycin and methylgriselimycin
WO2012024394A3 (en) Compositions comprising paulownin and/or paulownia extracts and uses thereof
WO2012064743A3 (en) Methods for improving heart function

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 11815240

Country of ref document: EP

Kind code of ref document: A2

NENP Non-entry into the national phase

Ref country code: DE

122 Ep: pct application non-entry in european phase

Ref document number: 11815240

Country of ref document: EP

Kind code of ref document: A2